Novel Drugs Summary
U.S. Food and Drug Administration
Center for Drug Evaluation and Research
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Welcome to the FDA’s Center for Drug Evaluation and Research’s (CDER’s) sixth annual Novel Drugs Summary.
Each year, CDER approves hundreds of new medications, most of which are variations of previously existing products, such as important new dosage forms of already-approved products, or cost-saving generic formulations. These new products contribute to quality of care, greater access to medication, more consumer choice, and a competitive marketplace that enhances affordability and public health. However, a small subset of these new approvals, referred to as novel drugs, are among the more innovative products that often help advance clinical care to another level. At the end of each calendar year, CDER summarizes these new products.
Our annual summary reports the number of novel drugs approved. However, we also focus on the medical value of many of these new drugs, their contributions to enhanced patient care, and the various regulatory tools CDER used to help ensure their safe and efficient development and approval. In 2016, novel approvals include the first treatment for patients with spinal muscular atrophy, the first drug approved to treat Duchenne muscular dystrophy, a new drug to treat hallucinations and delusions in some people with Parkinson’s disease, a new drug to treat patients with a rare chronic liver disease known as primary biliary cirrhosis, and two new treatments for patients with hepatitis C. The field also includes new treatments for patients with ovarian cancer, bladder cancer, soft tissue sarcoma, and chronic lymphocytic leukemia—as well as two new diagnostic agents for detecting certain forms of cancer.
All of these newly approved products were required to meet our rigorous premarket safety standards—and they will all be part of a strong postmarket safety surveillance system watching how they perform after they are more widely used by larger patient populations. Complementing this year’s summary of novel approvals is CDER’s recent report titled, Drug Safety Priorities 2015-2016, which details the Center’s key safety priorities as well as the depth and versatility of drug safety initiatives across CDER and the FDA. The report includes program updates and milestones achieved since the start of 2015, describing a variety of the FDA’s most important efforts in drug safety science, surveillance, and oversight.
We hope our Novel Drugs summary provides an appreciation of the expected impact that many of the novel drug approvals of 2016 will have on patient care, as well as the valuable role CDER played in helping to bring these drugs to market.
Janet Woodcock, M.D.
Director, Center for Drug Evaluation and Research
CDER’s 2016 Novel Drug Approvals
22 novel drugs
In calendar year 2016, FDA’s Center for Drug Evaluation and Research (CDER) approved 22 novel drugs, approved either as new molecular entities (NMEs) under New Drug Applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs). Listed below are CDER’s novel drugs of 2016.*
Novel drugs are often innovative products that serve previously unmet medical needs or otherwise significantly help to advance patient care and public health. NMEs have chemical structures that have never been approved before. However, in some cases an NME may have actions similar to earlier drugs and may not necessarily offer unique clinical advantages over existing therapies. This report summarizes all of the 2016 NME and novel BLA approvals, emphasizing those that offer new and innovative treatments to patients in need.
The vertical bars in the graph below indicate the number of novel drugs approved by CDER in each year of the past decade. CDER approved 22 novel drugs in 2016. From 2007 through 2015, CDER has averaged about 30 novel drug approvals per year.
Applications for new approvals remain steady
CDER approved a lower than average number of novel drugs in 2016, but the number of applications for these drugs that sponsors have submitted over time has remained relatively stable.
The points connected by lines in the graph below indicate the number of new NDA and BLA applications for NMEs and new therapeutic biologics CDER has received and filed for approval during the last 10 years. From 2007 through 2015, CDER filed an average of about 36 applications for novel drugs per year. CDER estimates 41 filings for 2016, which is consistent with previous years in this decade.
Novel Drugs Approved by CDER in Calendar Year 2016 (see New Molecular Entity and Therapeutic Biologics of 2016 for their non-proprietary names, approval dates, and what they are used for.)
Adlyxin, Anthim, Axumin, Briviact, Cinqair, Defitelio, Epclusa, Eucrisa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Taltz, Tecentriq, Venclexta, Xiidra, Zepatier, Zinbryta, Zinplava.
* This information is accurate as of December 31, 2016. 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.
- In 2016 CDER approved 22 novel drugs
- 22 novel drug approvals in CY 2016 is less than the average number approved annually during the past decade
- From 2007 through 2015 CDER averaged about 30 novel drug approvals per year
*The 2016 filed numbers include those filed in CY 2016 plus those currently pending filing (i.e., within their 60 day filing period) in CY 2016.
- Receipts that received a “Refuse to File” (RTF) or “Withdrawn before filing” (WF) identifier are excluded.
- Multiple submissions (multiple or split originals) pertaining to a single new molecular/biologic entity are only counted once.
- The filed number is not indicative of workload in the PDUFA V Program.
Impact on Public Health
Many of the 22 novel drugs CDER approved in 2016 are notable for their potential positive impact and unique contributions to quality medical care and public health.
CDER identified eight of the 22 novel drugs approved in 2016 (36%) as First-in-Class, which is one indicator of the innovative nature of a drug. These drugs often have mechanisms of action different from those of existing therapies.
Defitelio, Exondys 51, Ocaliva, Spinraza, Venclexta, Xiidra, Zinbryta, Zinplava
Noteworthy First-in-Class products include:
Defitelio - To treat adults and children who develop hepatic veno-occlusive disease with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called hematopoietic stem cell transplantation
Zinbryta - To treat multiple sclerosis
Drugs for Rare Diseases
Nine of the 22 novel drugs approved in 2016 (41%) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans. This is significant because patients with rare diseases often have few or no drugs available to treat their conditions.
Anthim, Defitelio, Exondys 51, Lartruvo, Netspot, Ocaliva, Rubraca, Spinraza, Venclexta
Noteworthy examples of drugs to treat rare diseases among the 2016 novel drugs include:
Exondys 51 - To treat patients with Duchenne muscular dystrophy
Spinraza - For treatment of patients with spinal muscular atrophy
Other Noteworthy Novel Drugs of 2016
In addition to the noteworthy examples of innovative First-in-Class and “orphan” new products mentioned above, the 2016 novel drug field also includes a variety of other notable drugs. These include cancer therapies, Lartruvo to treat patients with a form of cancer called soft tissue sarcoma; Rubraca, to treat women with ovarian cancer; Tecentriq, to treat patients with the most common type of bladder cancer (urothelial carcinoma), and Venclexta, for certain patients with chronic lymphocytic leukemia. Also notable are two diagnostic agents, Axumin, which is an imaging agent to help detect prostate cancer, and Netspot, another imaging agent used to detect rare neuroendocrine tumors.
This year’s novel approvals also include two new treatments for hepatitis C—Epclusa, to treat all six major forms of hepatitis C virus; and Zepatier, to treat adult patients infected with chronic hepatitis C virus genotypes 1 and 4.
Additional noteworthy approvals include Nuplazid, to treat hallucinations and delusions associated with psychosis experienced by some people with Parkinson’s disease, and Ocaliva, to treat patients with a rare chronic liver disease known as primary biliary cirrhosis.
Methods for expediting innovative novel drugs to market
CDER used a number of regulatory methods to expedite the development and approval of novel drugs in 2016, including: Fast Track, Breakthrough, Priority Review, and Accelerated Approval.
Fast Track drugs have the potential to address unmet medical needs. Eight of the 2016 novel drugs (36%) were designated by CDER as Fast Track. Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to 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: Anthim, Defitelio, Epclusa, Exondys 51, Lartruvo, Ocaliva, Spinraza, Zinplava
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. CDER designated seven of the 2016 novel drugs (32%) as Breakthrough therapies. A breakthrough therapy designation includes all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough status is designed to help shorten the development time of a potential new therapy.
Drugs designated with Breakthrough status were: Epclusa, Lartruvo, Nuplazid, Rubraca, Tecentriq, Venclexta, Zepatier
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. Fifteen of the 2016 novel drugs (68%) were designated Priority Review.
Drugs designated with Priority Review status were: Axumin, Defitelio, Epclusa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Tecentriq, Venclexta, Xiidra, Zepatier, Zinplava
The Accelerated Approval program allows for early approval of a drug for serious or life threatening illness that offers a benefit over current treatments. CDER approved six of the 2016 novel drugs (27%) under the Accelerated Approval program. This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that we consider reasonably likely to predict a clinical benefit of the drug. Once Accelerated Approval is granted, the drug must undergo additional testing to confirm that benefit. This speeds the availability of the drug to patients who need it.
Drugs that received Accelerated Approval were: Exondys 51, Lartruvo, Ocaliva, Rubraca, Tecentriq, Venclexta
Overall use of expedited development and review methods
Sixteen of the 2016 novel drugs (73%) were designated in one or more expedited categories of Fast Track, Breakthrough, Priority Review, and/or Accelerated Approval. Each of these designations helps expedite the speed of the development and/or approval process and is designed to help bring important medications to the market as quickly as possible.
Drugs that used at least one expedited review program include: Anthim, Axumin, Defitelio, Epclusa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Tecentriq, Venclexta, Xiidra, Zepatier, Zinplava
Fast Track 36%
Priority Review 68%
Accelerated Approval 27%
One or more expedited development and review methods 73%
PDUFA Goal Dates Met
Under the Prescription Drug User Fee Act (PDUFA), sponsors are assessed user fees that provide FDA with the additional resources needed to meet performance goals. Throughout the year, CDER was able to meet or exceed most PDUFA goal dates for application review, agreed to with the pharmaceutical industry and approved by Congress. In 2016, CDER met its PDUFA goal dates for 95% of the novel drugs approved (21 of 22).
Drugs for which CDER met our PDUFA review goal include: Adlyxin, Anthim, Axumin, Briviact, Cinqair, Defitelio, Epclusa, Eucrisa, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Taltz, Tecentriq, Venclexta, Xiidra, Zepatier, Zinbryta, Zinplava
In 2016, CDER met its PDUFA goal for 95% of the novel drugs approved in 2016
First Cycle Approval
CDER approved 21 of the novel drugs of 2016 (95%) on the “first cycle” of review, meaning without requests for additional information that would delay approval and lead to another cycle of review.
Drugs approved on the first cycle include: Adlyxin, Anthim, Axumin, Briviact, Cinqair, Defitelio, Epclusa, Eucrisa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Taltz, Tecentriq, Venclexta, Zepatier, Zinbryta, Zinplava
Approval in the United States Before Other Countries
Comparing approval to other countries offers another measure of approval efficiency. Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 19 of the 22 novel drugs approved in 2016 (86%) were approved in the United States before receiving approval in any other country.
Drugs approved first in the U.S. include: Anthim, Axumin, Cinqair, Epclusa, Eucrisa, Exondys 51, Lartruvo, Netspot, Nuplazid, Ocaliva, Rubraca, Spinraza, Taltz, Tecentriq, Venclexta, Xiidra, Zepatier, Zinbryta, Zinplava
First Cycle Approval 95%
Approved First in U.S. 86%
This document represents a broad overview of CDER approvals of novel drugs for calendar year 2016.
A continuing upward trend for the annual number of CDER’s novel drug approvals relies on a corresponding increase in the number of drug applications submitted for approval. During the past decade, submissions of applications for NMEs and novel BLAs by the pharmaceutical and biotechnology industry have remained relatively stable.
More important than the quantity of novel drugs approved in 2016 are the qualities of the new drugs the pharmaceutical industry has developed and the important new roles these drugs are serving to advance medical care.
Also noteworthy is the efficiency with which most of these drugs were reviewed and approved. CDER used a variety of expedited development and review regulatory tools in an effort to help speed these drugs to market.
In all cases, while striving for efficiency of review and approval of applications for new drugs, CDER maintains its rigorous standards for demonstration of effectiveness and safety in the process.
More important than the quantity of novel drugs approved by CDER in 2016 is their medical value and the important new roles they are serving to advance patient care.