CENTER FOR DRUG EVALUATION AND RESEARCH

ADVANCING HEALTH
THROUGH INNOVATION
2017 NEW DRUG
THERAPY APPROVALS
Impact | Innovation | Predictability | Access | Engagement

January 2018
www.fda.gov
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Advancing Health Through Innovation

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Table of Contents
Introduction ............................................................................................................................ 5

2017: A Year of Innovation and Advances ................................................................... 6

CDER’s Drug Therapy Approvals of 2017 ...................................................................... 8

Novel Drugs ............................................................................................................................ 8

Impact of Novel Drug Approvals .......................................................................... 10
Innovation: Frequent Use of Expedited Development and Review Pathways .... 13
Predictability: Meeting PDUFA Goals .................................................................. 14
Access: First Cycle Approval and Approvals Compared to Other Countries ....... 15

New and Expanded Uses of Already FDA-Approved Drugs ................................. 17

New Uses ............................................................................................................... 17
New Populations.................................................................................................... 20

Additional Approvals .......................................................................................................... 21

Biosimilars............................................................................................................. 21
New Formulations ................................................................................................ 22
Other New Formulations and Notable Approvals ................................................ 23
New Dosage Forms................................................................................................ 26

Ensuring Access to CDER-Regulated Products in an Emergency......................... 27

External Engagement .......................................................................................................... 29

Medical and Scientific Engagement ...................................................................... 29
The Voice of the Patient ........................................................................................ 31

Conclusion ............................................................................................................................... 32

Appendix A: Drug Designation Summary.................................................................... 34

2017 New Drug Therapy Approvals

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Advancing Health Through Innovation

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Introduction
Welcome to our new report, Advancing Health through Innovation: New
Drug Approvals and Other Drug Therapy Advances of 2017. Every year
since 2012, FDA’s Center for Drug Evaluation and Research (CDER) has
issued our Novel Drugs summary, a report that helps illustrate CDER’s
role in bringing innovative new drug therapies that are safe and effective
to patients in need. This year, we have expanded the report to provide even
more valuable information.

Novel drugs can represent important new therapies for advancing patient
care. As you will see in this report, 2017 was no exception. There is,
however, more to the story. Our report this year also highlights new
approvals for drugs that are not novel, yet offer significant clinical advances
that provide important medical value. As in other years, many important
advances in 2017 are approvals to use an already FDA-approved drug for a
Janet Woodcock, M.D.
new purpose or to treat a new population of patients, such as children.
Director, Center for Drug
Our report also emphasizes the many innovative regulatory tools CDER Evaluation and Research
uses to enhance our efficiency and expedite the review and approval of drug
therapies never marketed in the United States. It is important to recognize
that the decisions we made on these approvals were completed by or before
their goal dates as defined by Congressionally-approved agreements with
industry (referred to as user fee programs), and that most were approved
in the United States before any other country in the world.

CDER also played an active role in FDA’s response to the severe

hurricanes of 2017 by maintaining communications with pharmaceutical
manufacturers, identifying potential shortages of key medical products and
working closely with manufacturers to address these shortages, conducting
expedited reviews, and utilizing enforcement discretion to mitigate drug
shortages of medically necessary products.

Importantly, CDER cannot effectively evaluate the safety and effectiveness

of all of these new drug therapies without a great deal of help from a wide
range of stakeholders throughout manufacturing, scientific, medical, and
patient-focused organizations. We take the opportunity in this report to
highlight some of the many public-private partnerships and consortia
CDER leads or participates in to support innovation and improve health
care. The role of the patient in drug development and approval is becoming
increasingly vital. Our report also shares some of the important progress
FDA has made in incorporating the patient perspective into our decision-
making.

We hope our new report provides a deeper understanding of the many

ways CDER works to support innovation and improve treatments for
patients.

2017 New Drug Therapy Approvals

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2017: A Year of
Innovation and Advances
In 2017, FDA’s Center for Drug Evaluation and Research’s (CDER’s)
new drug therapy approvals helped a wide range of patients suffering
from many different medical conditions—from rare disorders to
common diseases—to gain new hope for improved quality of life, and in
some cases, improved chances of surviving life-threatening illnesses.

Rare Diseases
Among many other new approvals to help patients with rare diseases,
CDER approved the first new treatment for patients with sickle cell
disease in almost 20 years and the first-ever non-blood product to
treat patients with hemophilia A with inhibitors. For the first time,
a treatment is available for adults diagnosed with giant cell arteritis,
a rare condition that results in inflammation of blood vessels. CDER
Many new treatment options
also approved a new treatment for the rare condition known as Batten
for patients in need. disease, which can cause seizures, dementia, and a variety of other
debilitating symptoms.

Advancing Health Through Innovation

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Infectious Diseases
We approved a new antibiotic to treat certain types of serious skin
infections, and another to treat complicated urinary tract
infections, including kidney infections. We also approved two new
treatments for certain patients with chronic hepatitis C; a new drug
to help prevent cytomegalovirus infection in patients who have
received a bone marrow transplant; and, the first therapy in the United
States to treat Chagas disease, a rare parasitic disease which, after
years of infection, can cause serious heart illness.

Neurological Disorders
Last year was a particularly productive year for approving new therapies
for patients with neurological disorders. CDER approved new therapies
to treat patients with tardive dyskinesia, a frequent side effect of
psychiatric medications, myasthenia gravis, a rare neuromuscular
disease, and new treatments for Duchenne muscular dystrophy,
for certain forms of multiple sclerosis, for amyotrophic lateral
sclerosis (often called Lou Gehrig’s disease), and for Parkinson’s
disease.

Cancer Therapies
2017 was also another strong year for making new cancer therapies CDER’s drug therapy approvals
available to patients in need. Among others, we approved new therapies of 2017 will help a wide range
for certain patients with acute lymphoblastic leukemia; Merkel
of patients suffering from many
cell carcinoma; certain forms of relapsed or refractory acute
myeloid leukemia; certain forms of lymphoma; recurrent different diseases.
epithelial ovarian, fallopian tube, or primary peritoneal
cancer; and specific forms of liver, breast, and colorectal cancer.
We also approved the first cancer treatment based on a genetic feature
of a cancer rather than the location of the body where the tumor
originated.

Other Advances
Also in 2017, CDER approved a new therapy for decreasing heart risk
for patients with diabetes, a new drug to treat adults with moderate-
to-severe eczema and three therapies to treat patients with moderate-
to-severe plaque psoriasis. Two of these were for adults and one was
for adolescents. We also approved the first drug in the United States
with a sensor embedded in the pill that records that the medication
was taken. And in response to the devastation inflicted by the 2017
Hurricane Season, CDER worked with pharmaceutical manufacturers
with facilities in affected areas to address potential drug shortages.

2017 New Drug Therapy Approvals

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CDER’s Drug Therapy

Approvals of 2017
In 2017, CDER approved a wide variety of drug therapies to improve
the health of the American public, including:

• Novel drugs, which are often among the more innovative

products in the marketplace, and/or help advance clinical
care by providing therapies never before marketed in the
United States;

• New and expanded uses for already FDA-approved drugs;

• Biosimilars, which are highly similar to already FDA-

approved therapeutic biological products. These approvals
add consumer choice and marketplace competition;

• New formulations or new manufacturers of already

FDA-approved products that can provide advantages over
original products, such as being able to take the drug on an
empty stomach instead of with food, and;

• New dosage forms that can add value to already FDA-

As with all approved drugs, such as chewable tablets for patients
FDA-approved products, unable to swallow pills.

the new drug therapies This report summarizes these approvals and highlights
examples, emphasizing those approvals that offer new and
approved by CDER innovative treatments to patients in need.
discussed in this report
are associated with risks.
For more information
about these drugs Novel Drugs
and for complete risk Novel drugs are often innovative products that serve previously unmet
information, see the medical needs or otherwise significantly help to advance patient
treatments. The active ingredient or ingredients in a novel drug have
drugs’ approval letters never before been approved in the United States. This report lists
and FDA-approved all of CDER’s novel drug approvals of 2017 and also discusses those
that CDER considers notable advances. In 2017, CDER approved 46
labeling at
novel drugs, either as new molecular entities (NMEs) under New Drug
Drugs@FDA. Applications (NDAs), or as new therapeutic biologics under Biologics
License Applications (BLAs).

Novel Drug Approvals

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CDER’s Novel Drug Approvals of 2017

CDER’s novel drug approvals for 2017 are listed below.* See CDER’s Novel Drug Approvals for 2017 on the FDA
website for the approval dates, non-proprietary names, and what each drug is used for.

Aliqopa Brineura Imfinzi Ocrevus Solosec Vosevi

Alunbrig Calquence Ingrezza Ozempic Steglatro Vyzulta
Austedo Dupixent Kevzara Parsabiv Symproic Xadago
Bavencio Emflaza Kisqali Prevymis Tremfya Xermelo
Baxdela Fasenra Macrilen Radicava Trulance Xepi
benznidazole** Giapreza Mavyret Rhopressa Tymlos Zejula
Besponsa Hemlibra Mepsevii Rydapt Vabomere
Bevyxxa Idhifa Nerlynx Siliq Verzenio

* This information is accurate as of December 31, 2017. In rare instances, it may be necessary for FDA to change a drug’s NME designation
or the status of its application as a novel BLA. For instance, new information may become available which could lead to a reconsideration
of the original designation or status. If changes must be made to a drug’s designation or the status of an application as a novel BLA, the
Agency intends to communicate the nature of, and the reason for, any revisions as appropriate.
** Product approved with no trade name.

CDER’s Annual Novel Drug Approvals: 2008 - 2017

In 2017, CDER approved 46 novel drugs. The ten-year graph below shows that from 2008 through 2016, CDER has
averaged about 31 novel drug approvals per year.

50

45

45 46
Total Number of Novel Drugs Approved

40
41
35 39
30

25 30
26 27
20 24 21 22
15

10

0
2008 2009 2010 2011 2012 2013 2014 2015 2016 2017

Novel Drug Approvals

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Impact of Novel Drug Approvals

Many of the novel drugs CDER approved in 2017 are notable for their
potential positive impact and unique contributions to quality medical care
and patient treatment.

First-in-Class
Drugs designated First-in-Class
CDER identified 15 of the 46 novel drugs approved in 2017 (33%) as first-
and those approved to treat in-class, which is one indicator of the drug’s potential for strong positive
patients with rare diseases often impact on the health of the American people. These drugs often have
have a strong positive mechanisms of action different from those of existing therapies. Novel
drugs approved in 2017 identified as first in class by FDA were: Besponsa,
impact on patient health. Brineura, Dupixent, Emflaza, Giapreza, Hemlibra, Idhifa, Macrilen,
Mepsevii, Ocrevus, Prevymis, Radicava, Rhopressa, Rydapt, and Xermelo.

Examples of notable novel First-in-Class approvals for 2017 include:

• Dupixent (dupilumab) to treat adults with moderate-to-

severe eczema (atopic dermatitis), and;

• Ocrevus (ocrelizumab) to treat adults with relapsing forms of

multiple sclerosis and primary progressive multiple sclerosis.

Drugs for Rare Diseases

In 2017, 18 of CDER’s 46 novel drugs (39%) were approved to treat rare or
“orphan” diseases that affect 200,000 or fewer Americans. Patients with
rare diseases often have few or no drugs available to treat their conditions.
Novel drugs approved in 2017 with the orphan drug designation were:
Aliqopa, Alunbrig, Austedo, Bavencio, benznidazole, Besponsa, Brineura,
Calquence, Emflaza, Hemlibra, Idhifa, Macrilen, Mepsevii, Prevymis,
Radicava, Rydapt, Xermelo, and Zejula.

Examples of drugs that advance the care of patients with rare diseases
approved in 2017 include:

• Brineura (cerliponase alfa), a treatment for a specific

form of Batten disease, a rare disease that can cause
progressive neurological impairments, including seizures,
visual problems/blindness, personality and behavior
changes, dementia, and loss of the ability to walk, talk, and
communicate, and;

• Hemlibra (emicizumab), for the prevention of bleeding or to

reduce the frequency of bleeding episodes in patients with
hemophilia A who have developed antibodies called Factor
VIII inhibitors. This is the first non-blood product approved for
this condition.

Novel Drug Approvals

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Other Novel Drug Approvals: Advances in Patient Care Across a Broad Range of Diseases
In addition to the noteworthy first-in-class and orphan-designated drugs mentioned above, the 2017 novel drug field
also includes these notable examples --- approved for the first time in the United States, and likely to significantly
improve the care of patients with the diseases noted below:

A wide range of notable novel approvals in 2017 helps to enhance public health in the United States.

• Aliqopa (copanlisib) to treat adults with relapsed follicular lymphoma --- a slow-growing type of non-
Hodgkin lymphoma (a cancer of the lymph system) --- who have received at least two prior systemic
therapies;

• Bavencio (avelumab) for the treatment of patients 12 years and older with a rare and aggressive form of
cancer called metastatic Merkel cell carcinoma, including those who have not received prior
chemotherapy;

• Benznidazole to treat children ages 2 to 12 years with Chagas disease, a parasitic infection that can
cause serious heart illness after years of infection, and can also affect swallowing and digestion. This is the
first treatment approved in the United States for this rare disease;

• Besponsa (inotuzumab ozogamicin) for the treatment of adults with a type of cancer of the blood called
relapsed or refractory B-cell precursor acute lymphoblastic leukemia;

• Calquence (acalabrutinib) to treat adults with mantle cell lymphoma who have received at least one
prior therapy. Mantle cell lymphoma is a particularly aggressive cancer;

• Emflaza (deflazacort) to treat patients age 5 years and older with Duchenne muscular dystrophy, a rare
genetic disorder that causes progressive muscle deterioration and weakness;

• Giapreza (angiotensin II), for the treatment of hypotension in adults with distributive or vasodilatory
shock (dangerously low blood pressure despite adequate heart function) whose blood pressure remains
low despite receiving fluids and treatment with drugs called vasopressors;

• Idhifa (enasidenib) for the treatment of adults with relapsed or refractory acute myeloid leukemia, a
form of blood cancer, who have a specific genetic mutation;

• Ingrezza (valbenazine) to treat adults with tardive dyskinesia, a side effect of some antipsychotic
medications whereby patients can experience uncontrollable stiff, jerky movements of their face and
body, and other uncontrolled movements such as eye-blinking, sticking out the tongue, and arm-waving;

• Mavyret (glecaprevir and pibrentasvir) to treat adults with chronic hepatitis C virus genotypes 1-6
without cirrhosis (liver disease) or with mild cirrhosis, including patients with moderate to severe kidney
disease, as well as those who are on hemodialysis;

• Mepsevii (vestronidase alfa-vjbk) to treat patients with Sly syndrome or mucopolysaccharidosis type 7 –
a rare genetic disorder where an enzyme deficiency results in skeletal abnormalities, developmental delay,
enlarged liver and spleen, and narrowed airways, which can lead to respiratory infections;

Novel Drug Approvals

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• Nerlynx (neratinib) for the extended adjuvant treatment --- a form of therapy administered after an initial
treatment to further lower the risk of the cancer coming back --- of early-stage, human epidermal growth
factor receptor 2 (HER2)-positive breast cancer;

• Prevymis (letermovir) for prevention of an infection called cytomegalovirus (CMV) in patients who are
receiving a bone marrow transplant. CMV disease can cause serious health issues in these patients;

• Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis, commonly referred to as Lou
Gehrig’s disease, a rare disease that attacks and kills the nerve cells that control voluntary muscles;

• Rydapt (midostaurin) to treat adults newly diagnosed with a form of blood cancer known as acute
myeloid leukemia who have a specific genetic mutation called FLT3, in combination with chemotherapy;

• Siliq (brodalumab) to treat adults with moderate-to-severe plaque psoriasis, a chronic disorder in which
the body’s immune system sends out faulty signals that speed growth of skin cells that then accumulate,
causing red, flaky patches that can appear anywhere on the body;

• Symproic (naldemedine) for the treatment of opioid-induced constipation in adults with chronic non-
cancer pain;

• Tremfya (guselkumab) for the treatment of adults with moderate-to-severe plaque psoriasis;

• Trulance (plecanatide) to treat adults with chronic idiopathic constipation, which is a persistent
condition of constipation due to unknown origin;

• Vabomere (vaborbactam and meropenem) for treatment of adults with complicated urinary tract
infections, including pyelonephritis (kidney infection) caused by bacteria;

• Verzenio (abemaciclib) to treat adults who have hormone receptor (HR)-positive, HER2-negative
advanced or metastatic breast cancer that has progressed after taking therapy that alters a patient’s
hormones (endocrine therapy);

• Vosevi (sofosbuvir/velpatasvir/voxilaprevir) to treat adults with chronic hepatitis C virus genotypes 1-6
without cirrhosis (liver disease) or with mild cirrhosis;

• Xadago (safinamide) as an add-on treatment for patients with Parkinson’s disease who are currently
taking levodopa/carbidopa and experiencing “off” episodes;

• Xermelo (telotristat ethyl) combined with somatostatin analog (SSA) therapy to treat adults with
carcinoid syndrome diarrhea that SSA therapy alone has inadequately controlled, and;

• Zejula (niraparib) for the maintenance treatment (intended to delay cancer growth) of adults with
recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, whose tumors have
completely or partially shrunk (complete or partial response, respectively) in response to platinum-based
chemotherapy.

Novel drugs are often innovative products that serve previously unmet medical needs.

Novel Drug Approvals

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Innovation: Frequent Use of Expedited Development and Review Pathways

CDER used several regulatory pathways to expedite the development and approval of novel drugs in 2017. These
pathways utilize a range of approaches that can enhance development efficiency and shorten timelines; these
approaches can include more interactions between CDER staff and drug developers, greater program design flexibility,
and shortened timelines for review of applications.

Fast Track
Fast Track designated drugs have the potential to address unmet medical needs. Eighteen of the 46 2017 novel drugs
(39%) were designated by CDER as Fast Track. Fast Track speeds new drug development and review, for instance, by
increasing the level of communication between FDA and drug developers, and by enabling CDER to review portions of
a drug application ahead of the submission of the complete application.

Drugs designated with Fast Track status were: Aliqopa, Bavencio, Baxdela, Bevyxxa, Emflaza, Idhifa, Ingrezza,
Mavyret, Mepsevii, Ocrevus, Prevymis, Rydapt, Solosec, Vabomere, Verzenio, Vosevi, Xermelo, and Zejula.

Breakthrough Therapy
Breakthrough therapies are drugs with preliminary clinical evidence demonstrating that the drug may result in
substantial improvement on at least one clinically significant endpoint (e.g., study result) over other available
therapies for serious or life-threatening diseases for which there is unmet medical need. CDER designated 17 of the
2017 novel drugs (37%) as breakthrough therapies. A breakthrough therapy designation includes all the Fast Track
program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough
therapy designation is designed to help shorten the development time of a potential new therapy.

Drugs designated with Breakthrough therapy status were: Alunbrig, Bavencio, Besponsa, Brineura,
Calquence, Dupixent, Hemlibra, Imfinzi, Ingrezza, Kisqali, Mavyret, Ocrevus, Prevymis, Rydapt, Verzenio, Vosevi, and
Zejula.

Priority Review
A drug receives a Priority Review if CDER determines that the drug could potentially provide a significant advance in
medical care. The drug is reviewed within six months instead of the standard 10 months. Twenty-eight of the 46 novel
drugs approved in 2017 (61%) were designated Priority Review. Note, in some instances, priority review is assigned
as a result of the sponsor redeeming a voucher for priority review under CDER’s Priority Review Voucher program,
which may mean the drug does not potentially provide a significant advance. Such drugs are not included in the list
below.

Drugs designated Priority Review were: Aliqopa, Alunbrig, Bavencio, Baxdela*, benznidazole, Besponsa,
Bevyxxa, Brineura, Calquence, Dupixent, Emflaza, Giapreza, Hemlibra, Idhifa, Imfinzi, Ingrezza, Kisqali, Mavyret,
Mepsevii, Ocrevus, Prevymis, Rydapt, Solosec*, Vabomere*, Verzenio, Vosevi, Xermelo, and Zejula.

* Baxdela, Solosec, and Vabomere received Priority Review as Qualified Infectious Disease Products (QIDPs) as authorized by the
Generating Antibiotics Incentives Now Act (GAIN Act), which provides incentives to help bring new antibiotics and other antimicrobials
to market. These products may or may not have otherwise received the priority review designation.

Novel Drug Approvals

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Accelerated Approval
CDER used at least one expedited
The Accelerated Approval program allows FDA more flexibility in
development and review method what endpoints can be used to approve a drug that offers a benefit
to speed approval for over current treatments for a serious or life threatening illness. These

61%
accelerated approval endpoints may include ones that show benefits
over a shorter duration of treatment (where longer term demonstration
of benefit is needed for full approval) or are considered as “reasonably
of all novel drugs likely” to predict that an important clinical benefit will be seen.
approved in 2017. Subsequent confirmatory trials must be conducted to support full
approval. CDER approved six of the 2017 novel drugs (13%) under the
Accelerated Approval program. The application of accelerated approval
brings drugs that can provide important advances to patients sooner
than with traditional approvals.

Novel drugs approved in 2017 that received the Accelerated

Approval designation were: Aliqopa, Alunbrig, Bavencio,
benznidazole, Calquence, and Imfinzi.

Overall Use of Expedited Development

and Review Methods
Twenty-eight of the 2017 novel drug approvals (61%) were designated
in one or more expedited categories of Fast Track, Breakthrough,
Priority Review, and/or Accelerated Approval.

Novel drugs approved in 2017 using at least one expedited

approval method were: Aliqopa, Alunbrig, Bavencio, Baxdela,
benznidazole, Besponsa, Bevyxxa, Brineura, Calquence, Dupixent,
Emflaza, Giapreza, Hemlibra, Idhifa, Imfinzi, Ingrezza, Kisqali,
Mavyret, Mepsevii, Ocrevus, Prevymis, Rydapt, Solosec, Vabomere,
Verzenio, Vosevi, Xermelo, and Zejula.

Predictability: Meeting PDUFA Goals

CDER met its target date for Under the Prescription Drug User Fee Act (PDUFA), sponsors are
assessed user fees that provide FDA with the additional resources

100% needed to maintain an efficient and effective review process.

Throughout the year, CDER met or exceeded every PDUFA goal date
of 2017’s novel for application review agreed to with the pharmaceutical industry and
drug approvals. approved by Congress. In 2017, CDER met its PDUFA goal dates for
100% of the novel drugs approved (46 of 46).

Novel Drug Approvals

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Access: First Cycle Approval and Approvals

Compared to Other Countries
First Cycle Approval
CDER approved 39 of the 46 novel drugs of 2017 (85%) on the “first
cycle” of review, meaning without requests for additional information
that would delay approval and lead to another cycle of review. From
2011 through 2016, CDER approved 204 novel drugs, of which 166
(81%) were approved on the first cycle. The rate for 2017 is consistent
with this average. This high proportion of first cycle approval reflects the
extensive discussions between CDER staff and drug developers that go
on during drug development. This helps to assure that the application
contains the information CDER needs to be able to fully review—and if
appropriate—approve an application.

Novel drugs approved in 2017 on the first cycle were: Aliqopa,

Alunbrig, Bavencio, Baxdela, benznidazole, Besponsa, Bevyxxa,
Brineura, Calquence, Dupixent, Emflaza, Fasenra, Giapreza, Hemlibra,
Idhifa, Imfinzi, Ingrezza, Kisqali, Mavyret, Mepsevii, Nerlynx, Ocrevus,
Ozempic, Prevymis, Radicava, Rhopressa, Rydapt, Siliq, Solosec,
Steglatro, Symproic, Tremfya, Trulance, Tymlos, Vabomere, Verzenio,
Vosevi, Xermelo, and Zejula.

Approval in the United States Before Other Countries

Although regulatory processes differ widely between FDA and those of
regulatory agencies in other countries, 36 of the 46 novel drugs
78%
of the novel drugs approved in
approved in 2017 (78%) were approved in the United States before
receiving approval in any other country. 2017 were approved first in the
United States.
Novel drugs of 2017 approved first in the United States were:
Aliqopa, Alunbrig, Austedo, Bavencio, Baxdela, Bevyxxa, Brineura,
Calquence, Dupixent, Fasenra, Giapreza, Hemlibra, Idhifa, Imfinzi,
Ingrezza, Kisqali, Macrilen, Mavyret, Mepsevii, Nerlynx, Ocrevus,
Ozempic, Prevymis, Rhopressa, Rydapt, Steglatro, Symproic, Tremfya,
Trulance, Tymlos, Vabomere, Verzenio, Vosevi, Vyzulta, Xermelo, and
Zejula.

See Appendix A for

a summary chart of
designations for CDER’s
novel drug approvals.

Novel Drug Approvals

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2017’s Novel Drug Approvals

Expedited Review Pathway Usage

Accelerated Fast
Approval (6 of 46) Track (18 of 46)
Aliqopa Ocrevus
Aliqopa benznidazole 13%
61%
Alunbrig Calquence Bavencio Prevymis
Bavencio Imfinzi Baxdela Rydapt
39% Bevyxxa Solosec
Emflaza Vabomere
Idhifa Verzenio

Priority Ingrezza Vosevi

Review (28 of 46) Mavyret Xermelo

Mepsevii Zejula
Aliqopa Imfinzi
Alunbrig Ingrezza Used One or More
Bavencio Kisqali Expedited Pathway Breakthrough
Baxdela Mavyret (28 of 46) Therapy (17 of 46)
benznidazole Mepsevii
Besponsa Ocrevus
Aliqopa Emflaza Prevymis 37% Alunbrig Kisqali
Bevyxxa Prevymis
61% Alunbrig Giapreza Rydapt
Bavencio Mavyret
Bavencio Hemlibra Solosec
Brineura Rydapt Besponsa Ocrevus
Baxdela Idhifa Vabomere
Calquence Solosec Brineura Prevymis
benznidazole Imfinzi Verzenio
Dupixent Vabomere Calquence Rydapt
Besponsa Ingrezza Vosevi
Emflaza Verzenio Dupixent Verzenio
Bevyxxa Kisqali Xermelo
Giapreza Vosevi Imfinzi Vosevi
Brineura Mavyret Zejula
Hemlibra Xermelo Hemlibra Zejula
Calquence Mepsevii
Idhifa Zejula Ingrezza
Dupixent Ocrevus

Other Key Measures

50

45
100%
Total Novel Drugs Approved in 2017

40
(46 of 46)
35 85%
(39 of 46) 78%
30
(36 of 46)
25

20

15 39%
10
33% (18 of 46)
(15 of 46)
5

0
Met PDUFA First-In-Class Drugs for First Cycle Approved First in
Goal Date Orphan Approval U.S. Before Any
Diseases Other Country

Novel Drug Approvals

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New and Expanded
Uses of Already
FDA-Approved Drugs

After CDER approves a new drug, it

is not uncommon for a manufacturer
to submit an application with new
data that demonstrate safety and
effectiveness of the same product for
an additional purpose or for use in a
different population of patients.

After CDER approves a new drug, it is not uncommon for a manufacturer to submit an application with new data that
demonstrate safety and effectiveness of the same product for an additional purpose or for use in a different population
of patients. Applications to modify the use of an already-approved drug or to expand its use to other patients are in a
category of supplemental applications known as “efficacy supplements.”

New Uses
The products below are some notable approvals of 2017 for new uses of an already-FDA-approved drug:

• Actemra (tocilizumab), originally approved in 2010 to treat patients with rheumatoid arthritis. It was
approved in May 2017 for a new use to treat adults diagnosed with giant cell arteritis, a form of vasculitis,
which is a group of disorders that results in inflammation of blood vessels. This is the first FDA-approved
therapy specifically for this type of vasculitis. In August 2017, Actemra was also approved to treat patients
2 years of age and older with severe or life-threatening complications of cytokine release syndrome, a
condition related to a reaction caused by a treatment called chimeric antigen receptor (CAR) T cell therapy;

• Dysport (abobotulinumtoxinA), first approved by FDA in 2009 to treat adults with cervical dystonia
(torticollis) and wrinkles between the eyebrows. It was approved in 2017 to treat muscle spasticity in
adults;

• Imbruvica (ibrutinib), originally approved in 2013 to treat patients with mantle cell lymphoma, a rare
and aggressive blood cancer. In 2017, FDA approved the drug for treatment of adults with chronic graft
versus host disease (cGVHD) after one or more treatments --- typically corticosteroids to suppress their
immune system --- have failed. This was the first therapy approved by FDA specifically to treat cGVHD, a
rare and life-threatening condition that can occur after an allogeneic (bone marrow or peripheral blood

New and Expanded Uses

 18

cells) transplant. Also of note, in 2017, CDER expanded the

approved use of Imbruvica as the first FDA-approved treatment
for certain patients with a blood cancer called marginal zone
lymphoma;

• Nucala (mepolizumab), originally approved in 2015 for use

with other asthma medicines for the maintenance treatment
of asthma in patients age 12 years and older. It was approved
in 2017 to treat patients with eosinophilic granulomatosis
with polyangiitis (EGPA), formerly known as Churg-Strauss,
an extremely rare disease for which there are only about two to
five new cases a year per one million people. EGPA is caused by
inflammation (swelling) that occurs in certain types of cells in
blood or in tissues, which causes injury to organ systems. The
most commonly involved areas are the lungs, nose, sinuses,
skin, joints, nerves, intestinal tract, heart, and kidneys. This is
the first FDA-approved therapy specifically to treat EGPA;

• Opdivo (nivolumab), originally approved in 2014 to treat

patients with unresectable (cannot be removed by surgery) or
metastatic (advanced) form of skin cancer called melanoma
who no longer respond to other drugs. Since that approval,
it has been approved for a variety of other uses related to
cancer therapy. Notably, in 2017, Opdivo was approved for the
treatment of certain patients with hepatocellular carcinoma,
a type of liver cancer;

• Keytruda (pembrolizumab), originally approved in 2014 to

2017 marked the treat patients with advanced or unresectable melanoma who
first time FDA has approved are no longer responding to other drugs. Throughout 2014-
2017, it was approved for many new uses to treat patients with
a cancer treatment various forms of cancer. In May 2017, Keytruda was approved
based on a common to treat patients whose cancers have a specific genetic
feature (biomarker). This was the first time FDA has approved a
biomarker cancer treatment based on a common biomarker rather than
rather than the location of the body the location in the body where the tumor originated. In 2017,
CDER also expanded its approved use to include treatment of
where the tumor originated.
patients with refractory classical Hodgkin lymphoma, a
particularly difficult-to-treat type of Hodgkin lymphoma in
which the cancer returns after treatment. In 2017, Keytruda
was also approved to treat certain patients with recurrent,
locally advanced or metastatic forms of stomach cancer called
gastric or gastro-esophageal junction adenocarcinoma;

• Revlimid (lenalidomide), originally approved in 2005 to treat

certain patients who receive blood transfusions who have a
form of anemia called transfusion dependent anemia. It was
approved in 2017 as maintenance treatment for patients with
multiple myeloma (cancer of blood plasma cells) following

New and Expanded Uses

 19

a specific type of stem cell transplant intended to increase a

patient’s ability to produce new blood cells. This is the first FDA-
approved maintenance treatment for multiple myeloma;

• Soliris (eculizumab), first approved in 2007 to prevent the

breakdown of red blood cells in people with the rare disease
known as paroxysmal nocturnal hemoglobinuria, and
subsequently also approved in 2011 to treat patients with another
rare disease called atypical hemolytic uremic syndrome, a chronic
blood disorder. It was approved in 2017 to treat patients with
myasthenia gravis, a rare neuromuscular disease;

• Somatuline Depot (lanreotide) injection, originally approved in

2007 to treat acromegalic patients --- those with excessive growth
hormone in their body. In 2017, it was approved for the treatment
of adults with carcinoid syndrome --- a neurochemical imbalance
in the body caused by a cancerous tumor which can result in
diarrhea and skin flushing. Patients taking the drug required fewer
days on their rescue short-acting somatostatin analogs;

• Stelara (ustekinumab), first approved in 2009 to treat adults with

plaque psoriasis. It was approved in 2017 for the treatment of
adults with moderately to severely active Crohn’s disease who
have failed or were intolerant of other systemic treatments (see
also Stelara under New Populations);

• Sutent (sunitinib malate), first approved in 2006 to treat certain In 2017,

patients with gastrointestinal stromal tumors and advanced renal
cell carcinoma (both are forms of cancer). In 2017, it was approved CDER approved the
for the new use of the adjuvant treatment of adults with renal cell first treatment for
carcinoma, a type of kidney cancer, who are at a high risk of the
cancer returning after a kidney has been removed. Adjuvant
liver cancer in
treatment is a form of therapy that is taken after surgery to lower almost a decade.
the risk of the cancer returning. This new approval fills an unmet
medical need as it is the first adjuvant treatment approved for
patients with renal cell carcinoma;

• Stivarga (regorafinib), originally approved in 2012 to treat

patients with a certain form of colorectal cancer. It was approved
for a new use in April 2017 to include treatment of patients
with hepatocellular carcinoma (liver cancer) who have been
previously treated with the drug sorafenib. This was the first new
FDA-approved treatment for liver cancer in almost a decade;

• Victoza (liraglutide), originally approved in 2010 to treat patients

with type 2 diabetes. In 2017, CDER expanded the approved use
of the drug to include treatment for reducing risks of heart
attack, stroke and cardiovascular deaths in patients with type
2 diabetes, and;

New and Expanded Uses

 20

• Zelboraf (vemurafenib), first approved in 2011 to treat

certain patients with melanoma whose malignant cells have
a specific genetic mutation known as BRAF V600. In 2017
it was approved for the treatment of certain adults with
Erdheim-Chester Disease (ECD), a rare cancer of the blood.
Zelboraf is approved for this condition for patients whose
malignant cells harbor the BRAF V600 mutation. This the first
FDA-approved treatment for ECD.

Expanded approval can bring New Populations

treatment options to a much larger The products listed below are notable approvals in 2017 of an already-
approved drug for use in an expanded population of patients:
population of patients, such as
children with cystic fibrosis. • Kalydeco (ivacaftor), originally approved in 2012 to treat a
narrow population of pediatric patients with cystic fibrosis
(CF), was approved in 2017 to treat new populations of
children with one of 23 additional rare mutations. Kalydeco is
now indicated for 33 CF mutations, up from 10 previously;

• Sovaldi (sofosbuvir) and Harvoni (ledipasvir), first approved

in 2013 and 2014 respectively, were approved only to treat
adults infected with the hepatitis C virus (HCV). In 2017,
both drugs were approved to also treat children with HCV,
and;

• Stelara (ustekinumab), approved first in 2009 to treat

adults with plaque psoriasis. It was approved in 2017 to
treat adolescent patients 12 years or older, with moderate
to severe plaque psoriasis --- only the second systemic
biologic therapy approved for pediatric patients with
psoriasis (see also Stelara in New Uses).

New and Expanded Uses

 21

Additional Approvals
In addition to the many notable novel drug and efficacy supplement
approvals of 2017, CDER also approved a variety of other therapies.
Among these are biosimilars and drugs with new formulations,
manufacturers, combinations, or dosage forms of already FDA-
approved drugs, as well as others. Below discusses notable examples of
these various types of approvals.

Biosimilars
An FDA-approved biosimilar is highly similar to and has no clinically
meaningful differences in terms of safety, purity and potency (safety and
effectiveness) from an already FDA-approved biological product, called
the reference product. Biological products are highly complex, and often
used to treat patients with serious and life-threatening conditions. The law
allowing FDA to approve biosimilars was designed to create competition,
increase consumer choice, and support greater access to important
therapies.

In 2017, CDER approved five new biosimilars: Biosimilars expand treatment

• Cyltezo (adalimumab-adbm), biosimilar to Humira options and bring competition
(adalimumab), approved for the treatment of patients with to the U.S. marketplace.
rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing
spondylitis, psoriatic arthritis, Crohn’s disease, ulcerative
colitis, and plaque psoriasis;

• Ixifi (infliximab-qbtx), biosimilar to Remicade (infliximab),

which can be used to treat patients with rheumatoid arthritis,
psoriatic arthritis, ankylosing spondylitis, Crohn’s disease,
plaque psoriasis, and ulcerative colitis;

• Mvasi (bevacizumab-awwb), biosimilar to Avastin

(bevacizumab), used in the treatment of multiple types
of cancer, including treatment for certain patients with
metastatic colorectal cancer, non-squamous non-small cell
lung cancer, glioblastoma, metastatic renal cell carcinoma,
and cervical cancer;

• Ogivri (trastuzumab-dkst) biosimilar to Herceptin

(trastuzumab), for the treatment of patients with breast or
metastatic stomach cancer (gastric or gastroesophageal
junction adenocarcinoma) whose tumors overexpress the
HER2 gene, and;

• Renflexis (infliximab-abda), also biosimilar to Remicade

(infliximab) (see Ixifi above.).

Additional Approvals
 22

The first biosimilar in the United States, Zarxio (filgrastim-sndz), was

Many of CDER’s new drug approved in 2015 as a biosimilar to the brand name product, Neupogen
approvals are not novel drugs, but (filgrastim), which helps patients make white blood cells after being
depleted by a cancer treatment. In 2016, CDER approved three more
still offer important medical value
biosimilars: Inflectra (infliximab-dyyb), biosimilar to Remicade (see
to patients in need. Ixifi and Renflexis above); Erelzi (etanercept-szzs), biosimilar to Enbrel,
which treats autoimmune diseases, such as rheumatoid arthritis, by
interfering with a substance in the body called tumor necrosis factor; and
Amjevita (adalimumab-atto) biosimilar to Humira (see Cyltezo above).

Multiple biosimilars for each FDA-approved reference product can make

market competition even stronger. An increase in market competition
may lead to significantly reduced costs for both patients and our
healthcare system. The biopharmaceutical industry has shown great
interest in developing these products, and CDER expects many more
applications for biosimilars to be submitted. There are currently nine
FDA-approved biosimilars, but there are many more in the development
pipeline.

New Formulations
A new formulation of a drug is one in which the product’s active
ingredient is already FDA-approved. New formulations of already-
approved drugs can offer significant advances in therapy.

Opioids with Properties Intended to Deter Abuse

CDER approved three new formulations of already approved opioid
pain medications in 2017-- oxycodone, hydrocodone, and morphine
sulfate. These new formulations have properties that are intended
to deter abuse of these highly addictive medications. Although
these products are formulated to make abuse more difficult, they cannot
prevent abuse by all routes, nor are they expected to prevent or reduce
addiction. They joined seven other products approved previously by
FDA with properties intended to deter abuse. Given our nation’s ongoing
opioid epidemic, such new formulations have the potential to improve
public health. These three new approvals for 2017 are:

• Arymo ER (morphine sulfate extended-release tablets),

New opioid approvals of 2017 designed with inactive ingredients that make the tablet
may help to reduce abuse and more difficult to manipulate for misuse and abuse,
and thereby deter intravenous abuse. Arymo ER is the
contribute to efforts to address third morphine sulfate product approved by CDER with
our nation’s opioid epidemic. properties designed to deter abuse --- following the 2009
approval of Embeda and the 2015 approval of Morphabond;

Additional Approvals
 23

• Roxybond (oxycodone immediate-release tablets), the

first FDA-approved immediate-release opioid with
properties designed to deter abuse by the intravenous
and intranasal (snorting) routes. To date, CDER has
approved ten opioid products with properties intended
to deter abuse, but all except Roxybond are extended
release/long acting (ER/LA) preparations, and;

• Vantrela ER (hydrocodone extended-release tablets),

formulated with properties intended to make the tablet
more difficult to manipulate for misuse and abuse,
and deter abuse by the oral, intranasal (snorting) and
intravenous routes. Vantrela ER is the second ER/LA
hydrocodone product with properties intended to deter
abuse, after the 2014 approval of Hysingla (hydrocodone
ER).

All approved products with properties intended to deter abuse must

conduct post-approval epidemiologic studies to determine whether the
abuse-deterrent properties are actually having an effect on the rates of
abuse in the community.

Other New Formulations and Notable Approvals

Below includes other notable new formulations as well as other
notable non-novel drug approvals of 2017, including, but not limited
to, those with a new combination of active ingredients or a new
manufacturer of an already FDA-approved drug.

• Abilify MyCite (aripiprazole tablets with sensor). Abilify

was first approved by FDA in 2002, in tablet form, to treat
patients with schizophrenia. It was subsequently
approved to treat certain patients with bipolar I disorder
and as adjunctive treatment of major depressive disorder.
Generic versions have also since been approved and
aripiprazole is available in a variety of dosage forms: In 2017, CDER approved a
tablet, oral solution, and injectable, including extended tablet with an electronic sensor
release injections. In 2017, Abilify MyCite was approved as
a tablet that contains an electronic sensor that allows the that allows the patient to track
patient to track whether he or she has taken the whether he or she has
medication via a smartphone or the cloud. Patients can
also allow their caregivers or physician to access the taken the medication.
information through a web-based portal.

• Admelog (insulin lispro injection), a short-acting insulin

to help control blood glucose in patients 3 years and
older with type 1 diabetes mellitus and adults with
type 2 diabetes mellitus. It was approved through an
abbreviated pathway known as (505(b)(2), which allows

Additional Approvals
 24

a company to rely on FDA’s previous finding of safety

and effectiveness of already approved products if the
company has provided evidence that its product will
not differ substantially from the approved product. This
regulatory pathway may enable approval of therapies
in shorter amounts of time to help increase consumer
choice, create market competition, and possibly decrease
cost. This approval relied, in part, on comparison to the
already FDA-approved product, Humalog (insulin lispro
injection). Admelog is the first insulin lispro product and
the second insulin product approved via the 505(b)(2)
pathway, following the 2016 approval of Basalglar (insulin
glargine), approved based on comparison to the already
FDA-approved product, Lantus (insulin glargine).

• Endari (L-glutamine oral powder), the first treatment

In 2017, CDER approved the first
approved for patients with sickle cell disease in almost
treatment for patients with sickle 20 years. Prior to this approval, L-glutamine was approved
cell disease in almost 20 years. as a prescription drug in 2004 to treat certain patients
with short bowel syndrome. It has also been marketed as
a dietary supplement and used for treatments that include
digestive issues (e.g., stomach ulcers, ulcerative colitis,
Crohn’s disease), depression, moodiness, irritability,
anxiety, insomnia, and enhancing exercise performance;

• Juluca (rilpivirine and dolutegravir), a combination of

two FDA-approved drugs, each used in the treatment
of certain patients with HIV. It was approved in 2017
as a complete regimen for the treatment of certain
adults with HIV-1 infection. This is the first complete
regimen containing only two HIV-1 drugs and neither is
a nucleoside reverse transcriptase inhibitor (NRTI). Long-
term NRTI use is associated with some degree of kidney,
bone, and cardiovascular toxicities;

• Mylotarg (gemtuzumab ozogamicin), approved in

2017 for the treatment of adults with newly diagnosed
acute myeloid leukemia whose tumors express the
CD33 antigen (CD33-positive AML) and the treatment
of patients aged 2 years and older with CD33-positive
AML who have experienced a relapse or who have not
responded to initial treatment (refractory). Mylotarg
originally received accelerated approval in May 2000 as
a stand-alone treatment for older patients with CD33-
positive AML who had experienced a relapse, but was
voluntarily withdrawn from the market after subsequent
confirmatory trials failed to verify clinical benefit and
demonstrated safety concerns, including a high number
of early deaths. This new approval in 2017 was based on

Additional Approvals
 25

careful review of a new dosing regimen, which has shown

that the benefits of this treatment outweigh the risk;

• Noctiva (desmopressin), the first approval of desmopressin

to treat adults who wake up at least twice a night to
urinate because they overproduce urine, a condition
called nocturnal polyuria. Desmopressin was previously
approved by FDA for a different manufacturer to treat
diabetes insipidus and a variety of blood disorders;

• Rituxan Hycela (rituximab and hyaluronidase human),

a new combination of two already FDA-approved drugs. It
was approved in 2017 for the treatment of certain patients
with various forms of lymphoma (a type of blood cancer).
A dose of Rituxan Hycela can be administered in just a few
minutes compared to hours for a similar product, Rituxan
(rituximab), originally approved in 1997;

• Sublocade (buprenorphine), the first once-monthly In 2017, CDER approved the

injectable buprenorphine product to treat certain patients
with moderate-to-severe opioid use disorder. It is first once-monthly injectable
injected by a healthcare professional under the patient’s buprenorphine product to help
skin in the abdominal region and provides sustained
patients fighting opioid addiction.
plasma levels of buprenorphine over the dosing interval.
Buprenorphine was originally approved in 1981 and has
been used in a variety or other formulations to help
patients fighting opioid addiction;

• Symjepi (epinephrine), a product approved for a new Approval for a new manufacturer
manufacturer that enables a patient to self-inject
epinephrine to counteract a dangerous allergic reaction. of self-injectable epinephrine
There are other types of epinephrine products that are adds another choice for patients
intended for this use. This new product adds another
choice for patients and increases competition; and increases competition.

• Tepadina (thiotepa), a new formulation of the drug

thiotepa, a product approved by FDA since 1959 to treat
patients with a variety of cancers. Tepadina was approved
in 2017 for reducing the risk of graft rejection when
used in conjunction with certain other drugs to prepare for
a stem cell transplant. It is used to increase red blood cell
production, and;

• Vyxeos (cytarabine and daunorubicin), a combination

of two already FDA-approved drugs, approved for the
treatment of adults with two types of acute myeloid
leukemia (AML): newly-diagnosed therapy-related t-AML
or AML with myelodysplasia-related changes. This is the
first approved treatment specifically for patients with
certain types of high-risk AML.

Additional Approvals
 26

New Dosage Forms

New dosage forms can ensure a
New dosage forms for already FDA-approved drugs can improve
proper dose, help patients adhere
patient health by helping to increase patient adherence to therapy,
to treatment, and improve ensure a proper dose is taken, and improve quality of life for patients
quality of life. who must use the medication on a prolonged basis. Notable approvals
in this category include:

• Esbriet (pirfenidone) 534 mg. and 801 mg. tablets, new

dosage forms of a 267 mg. capsule originally approved
in 2014 as the first new drug in the United States to treat
patients with idiopathic pulmonary fibrosis. A typical
dose of the originally approved product is two or three
267 mg. capsules three times daily. The higher strength
tablets enable taking fewer tablets daily;

• Gocovri (amantadine extended release capsule),

approved by FDA in 2017 as a new dosage form of the
already FDA-approved drug amantadine, which has long
been approved to treat and prevent respiratory infections
caused by the influenza A virus, as well as to treat
symptoms of Parkinson’s disease and similar conditions,
such as stiffness and shaking. Gocovri is specifically
approved for the treatment of dyskinesia in certain
patients with Parkinson’s disease. It is the only FDA-
As with all approved medication for this use. Dyskinesia is a category
FDA-approved products, of movement disorders that are characterized by
involuntary muscle movements and diminished voluntary
the new drug therapies movements;
approved by CDER • Mydayis (mixed salts of a single-entity amphetamine), a
discussed in this report new amphetamine dosage form designed to enable once-
daily dosing and provide all day control for attention
are associated with risks. deficit hyperactivity disorder;
For more information
• Norvir (ritonavir) oral powder, a new dosage form of the
about these drugs tablet and oral solution forms of Norvir, an HIV protease
and for complete risk inhibitor approved in 1996 to be used in combination
with other antiretroviral agents for the treatment of HIV-1
information, see the infection. The oral powder formulation can be mixed
drugs’ approval letters in soft foods and does not contain ethanol or propylene
glycol and therefore may be a more palatable and safer
and FDA-approved alternative for children.
labeling at
Drugs@FDA.

Additional Approvals
 27

Ensuring Access to CDER-Regulated

Products in an Emergency
CDER’s drug therapy approvals discussed above were all conducted
under carefully planned and executed timelines --- with a goal of
bringing safe and effective new therapies to the American public as
soon as possible. But, when emergencies strike, CDER is flexible with
our use of enforcement discretion to help ensure access to all needed
medications for the protection of public health.

Hurricane Season 2017 caused widespread devastation to states along

the Gulf Coast, U.S. Caribbean island territories, and Caribbean island
nations. Well before Hurricanes Harvey, Irma, Jose, and Maria made
landfall, FDA was preparing for the impact on FDA-regulated products
by assessing types and locations of facilities in the paths of these
hurricanes and the effects of potential product shortages.
CDER’s responses during hurricane
When Hurricane Maria struck Puerto Rico, the storm caused a season of 2017 underscore
significant loss of manufacturing capability for many CDER-regulated
drug products made on the island. CDER staff worked tirelessly to help
the importance of emergency
these manufacturers mitigate potential drug shortages, using expedited access to FDA-approved medical
reviews and enforcement discretion for product importation, which products to protect public health.
helped facilitate availability of needed medications.

2017 New Drug Therapy Approvals

 28

Sodium chloride-based and glucose-based solutions for intravenous

infusion are critical for mixing drugs appropriately for use in hospitals
and medical clinics. A major manufacturer of these solutions, Baxter
Healthcare Corporation, lost much of its production capability for these
products after Maria struck. In response, CDER expedited review of
information on other Baxter manufacturing sites around the world, and
concluded that unapproved Baxter solutions could be imported and
distributed under enforcement discretion. CDER also allowed certain
waivers for approved drugs made by another company.

These steps helped to maintain supplies of important products for

treatment of serious conditions, such as cancers and autoimmune
diseases, and for prevention of organ rejection in kidney transplant
patients. Hurricane damage interrupted supplies of medical oxygen and
nitrogen, prompting CDER’s ongoing efforts with several companies to
help maintain supply.

Until generators could be turned on, the loss of electricity at

manufacturing plants in Puerto Rico exposed some drug products
to temperatures and humidity outside of their approved storage
CDER’s efforts after the hurricane conditions. Through interactions with certain companies, CDER
damage helped to maintain concluded that their manufacturing can resume in Puerto Rico when
power is restored or at other manufacturing sites. Although the
supplies of important products for
products made at locations experiencing power outages are not in
treatment of serious conditions. shortage, CDER will review plans for determining if product quality
was affected.

Advancing Health Through Innovation

 29

External Engagement
CDER collaborates with a wide variety of stakeholders including medical and scientific organizations and patient
advocacy groups, as well as patients, and caregivers, to support innovation and advance public health.

Medical and Scientific Engagement

CDER’s work with other government agencies, global organizations, academia, industry, and patient advocacy groups
helps to develop and advance the science needed to bring innovative new drug therapies to the marketplace. CDER
often facilitates the creation of partnerships and collaborations, and advises on specific scientific projects. Through
FDA’s Critical Path Initiative (CPI), CDER has initiated scores of scientific collaborations including the following:

• Improving drug testing by advancing the science of clinical trials, such as our work with:

• The Clinical Trials Transformation Initiative (CTTI);

• Enhancing the scientific techniques used to evaluate a drug’s safety and efficacy,
such as our work with:

• The Biomarkers Consortium (BC), and;

• The Global Pediatric Clinical Trials Network Pre-Launch Consortium (PTC).

External Engagement
 30

• Studying specific diseases and conditions where new therapies are particularly important for
public health, such as our work with:

• The Accelerating Medicines Partnership (AMP)

• The Analgesic Clinical Trial Translations, Innovations Opportunities and Networks Initiative (ACTTION)
• The Coalition Against Major Disease Consortium (CAMD)
• The Critical Path for Parkinson’s (CPP)
• The Critical Path to TB Drug Regimens Consortium (CPTR)
• The Duchenne Regulatory Science Consortium (D-RSC)
• The International Neonatal Consortium
• Kidney Health Initiative (KHI)
• The Multiple Sclerosis Outcome Assessments Consortium (MSOAC), and
• SmartTots.
• Developing common methods or standards, so scientists can better work together, such as our
work with:

• The Coalition For Accelerating Standards and Therapies (CFAST).

• Improving drug development by predicting safety earlier in product development, such as our
work with:

• The Cardiac Safety Research Consortium

• The HESI Cardiac Safety Technical Committee, and
• The Predictive Safety Testing Consortium (PSTC).
• Developing technology to assure quality in manufacturing and the drug supply chain, such as our
work with:

• The National Institute for Pharmaceutical Technology and Education (NIPTE)

• The Product Quality Research Institute (PQRI), and
• RX-360.
• Gaining information from the patient perspective, such as our work with:

• The Patient Reported Outcome Consortium (PRO), and

• Electronic Patient-Reported Outcome (ePRO) Consortium.

CDER also regularly engages with stakeholders via Critical Path Innovation Meetings to address issues in drug
development. These engagements spur development of tools such as animal- or computer-based predictive models,
biomarkers for safety and effectiveness, and new clinical methods.

These meetings have become a valuable tool by which CDER, industry, academia, patient advocacy groups, and other
government entities can communicate early in the drug development process to improve efficiency and success.

For more information on how CDER uses science to advance innovation, please see
Regulatory Science in Action on the FDA website.

External Engagement
 31

The Voice of the Patient

CDER’s external engagement also extends to our most important
stakeholders --- patients. Drug development and FDA’s review process
benefit when we can systematically obtain patients’ experiences,
perspectives, needs, and priorities about their condition and available
treatment options.

People living with a condition are uniquely positioned to inform our

understanding of the therapeutic context for drug development and
evaluation. Therefore, FDA is increasingly involving the patient in many
of our regulatory activities and decision-making processes.

Since 2013, our Patient Focused Drug Development (PFDD) initiative has Patient involvement has become
been helping FDA to better understand the needs of patients and how
they think and feel about new drug development. During this time, we
an integral part of the drug
have conducted 24 meetings with patients, caregivers, and patient development process.
advocacy groups.

The meetings have focused on disease areas for which there is a particular
need, in the context of drug development and evaluation, to better
understand patient perspective on their condition and treatments. In
2017, we conducted meetings to engage patients with sarcopenia, autism,
alopecia areata, and hereditary angioedema.

For each meeting, we publish a detailed Voice of the Patient report,

summarizing what we have learned. Completion of these meetings
was part of FDA’s commitment under the fifth authorization of the
Prescription Drug User Fee Act (PDUFA V).

We recognize that there are many more disease areas to address. To help
expand the benefits of FDA’s PFDD initiative, FDA encourages patient
organizations to identify and organize patient-focused collaborations
to generate public input on other disease areas, using the process
established through PFDD as a model. FDA recommends that patient
organizations who are interested in conducting an externally-led PFDD
meeting send FDA a Letter of Intent.

As we work to enhance patient-focused drug development and

regulatory decision-making, CDER recognizes the need for systematic,
methodologically sound approaches to collecting patient input in such
a way that the data can further inform regulatory decision-making.
An important step is to provide guidance to patient communities,
researchers, and drug developers on pragmatic and sound strategies,
pathways, and methods to gather and use patient input.

External Engagement
 32

Conclusion
CDER’s staff consists of individuals with a range of different areas of
expertise, including physicians, safety evaluators, chemists, biologists,
biostatisticians, nurses, pharmacists, pharmacologists, epidemiologists,
legal and regulatory experts, and many more --- working together
to bring safe and effective drug therapies to the American public as
efficiently as possible.

These therapies come in the form of novel drugs never before marketed
in the United States, other drugs that add important medical value,
already FDA-approved products approved for new uses and for
administration to new populations of patients, and new dosage forms of
products designed to offer advantages over earlier versions.
Although not a comprehensive
More important than the quantity of the new therapies is their medical
compilation of all our approvals value and the important new roles these drugs are serving to advance
for the year, this report serves to patient care.

provide a wide variety of valuable Also noteworthy is the efficiency with which these drugs were reviewed
examples of the many ways CDER and approved. CDER used a variety of expedited development and
regulatory review tools to help speed these drugs to market. Further, as
approves new drug therapies to illustrated by our response during the hurricane season of 2017, we
enhance patient health. expedited reviews and used importation under enforcement discretion
to facilitate access to needed medications.

Advancing Health Through Innovation

 33

In all cases, while striving for efficiency of review of applications

for new drug therapies, CDER maintains its rigorous standards for
demonstration of safety and efficacy.

Our drug therapy approvals of 2017 will help many patients in need for
years to come. However, CDER’s mission goes well beyond critically
reviewing the safety and efficacy of drug applications we receive from
industry. We also look to advance the science and technology that can
lead to future innovative drugs --- many of which may not yet even be
conceived. We are working to develop more efficient and innovative
approaches for evaluating the safety and efficacy of the drug therapies
that industry will develop with these new advances.

Although our regulatory work extends to many scientific, clinical, and

technological areas, we cannot accomplish all that is necessary on our
own. CDER works collaboratively with a wide range of stakeholders
across the medical community, including academia, industry, patients
and their caregivers, patient advocacy groups, state and other federal
agencies, and more. Listening has become an important component of
this work. We strive to ensure that we understand the needs of our key
constituencies and that we are providing the most benefit for patients
and the strongest possibilities for improved public health in America.

Of note, CDER works to assure that all of our approvals provide value to
the American public.

This report is not intended to be a comprehensive compilation of all

of CDER’s approvals, but rather to offer a wide variety of valuable
examples of the many ways CDER acts to enhance the health of the
American people.

2017 New Drug Therapy Approvals

 34

es
Appendix A:

Stat
val

al
Drug Designation Summary

ppro

prov

ited
oal

e Un
ed A
h

FA G

e Ap
ass

oug
l
-in-C

lerat

in th
PDU

Cycl
Trac

kthr
h an

rity
Approval Trade Active Dosage

First

Acce
Brea

First

First
Fast

M et
Prio
Or p
Date Name Ingredient Form

1/19/2017 Trulance plecanatide Tablet

2/7/2017 Parsabiv etelcalcetide Injection

Tablet,
2/9/2017 Emflaza deflazacort
Oral Solution

2/15/2017 Siliq brodalumab Injection

2/28/2017 Xermelo telotristat ethyl Tablet

3/13/2017 Kisqali ribociclib Tablet

3/21/2017 Xadago safinamide Tablet

3/23/2017 Bavencio avelumab Injection

3/23/2017 Symproic naldemedine Tablet

3/27/2017 Zejula niraparib Capsule

3/28/2017 Dupixent dupilumab Injection

3/28/2017 Ocrevus ocrelizumab Injection

4/3/2017 Austedo deutetrabenazine Tablet

valbenazine
4/11/2017 Ingrezza Capsule
tosylate

4/27/2017 Brineura cerliponase alfa Injection

4/28/2017 Alunbrig brigatinib Tablet

4/28/2017 Rydapt midostaurin Capsule

4/28/2017 Tymlos abaloparatide Injection

5/1/2017 Imfinzi durvalumab Injection

5/5/2017 Radicava edaravone Injection

5/22/2017 Kevzara sarilumab Injection

6/19/2017 Baxdela delafloxacin Tablet, Injection

6/23/2017 Bevyxxa betrixaban Capsule

Advancing Health Through Innovation

 35

es
Stat
val

al
ppro

prov

ited
oal

e Un
ed A
h

FA G

e Ap
ass

oug
l
-in-C

lerat

in th
PDU

Cycl
Trac

kthr
h an

rity
Approval Trade Active Dosage

First

Acce
Brea

First

First
Fast

M et
Prio
Or p
Date Name Ingredient Form

7/13/2017 Tremfya guselkumab Injection

7/17/2017 Nerlynx neratinib maleate Tablet

voxilaprevir;
7/18/2017 Vosevi velpatasvir; Tablet
sofosbuvir

8/1/2017 Idhifa enasidenib Tablet

glecaprevir;
8/3/2017 Mavyret Tablet
pibrentasvir
inotuzumab
8/17/2017 Besponsa Injection
ozogamicin

8/29/2017 benznidazole benznidazole Tablet

vaborbactam;
8/29/2017 Vabomere Injection
meropenem
copanlisib
9/14/2017 Aliqopa Injection
hydrochloride

9/15/2017 Solosec secnidazole Granules

9/28/2017 Verzenio abemaciclib Tablet

10/31/2017 Calquence acalabrutinib Capsule

11/2/2017 Vyzulta latanoprostene bunod Ophthalmic Solution

11/8/2017 Prevymis letermovir Tablet

11/14/2017 Fasenra benralizumab Injection

11/15/2017 Mepsevii vestronidase alfa-vjbk Injection

11/16/2017 Hemlibra emicizumab Injection

12/5/2017 Ozempic semaglutide Injection

12/11/2017 Xepi ozenoxacin Cream

12/18/2017 Rhopressa netarsudil Ophthalmic Solution

12/19/2017 Steglatro ertugliflozin Tablet

macimorelin
12/20/2017 Macrilen Oral Solution
acetate

12/21/2017 Giapreza angiotensin II Injection

2017 New Drug Therapy Approvals