Every year, the FDA’s Center for Drug Evaluation and Research (CDER) approves new medications. Some medications are variations of existing products, such as generic formulations or new dosage forms of previously-approved products. Other medications are novel drugs, having chemical structures that have never previously been approved for human use.

Novel Drug List

The CDER publishes a summary of the approved novel drugs on the FDA website. The FDA recently released the summary of the novel drugs approved in 2016 (click here for the full summary). The summary includes 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). The table below lists the 22 novel drugs approved in 2016 and related approval information.

No.

Drug Name

Active Ingredient

Approval Date

FDA-approved use

Pharmaceutical

Company

Special Designation

1.

Zepatier

elbasvir and grazoprevir

1/28/2016

To treat patients with chronic hepatitis C virus (HCV) genotypes 1 and 4 infections in adult patients.

Merck & Co. Inc.

2.

Briviact

brivaracetam

2/18/2016

To treat partial onset seizures in patients age 16 years and older with epilepsy.

UCB, Inc.

3.

Anthim

Obiltoxaximab

3/18/2016

To treat inhalational anthrax in combination with appropriate antibacterial drugs.

Elusys Therapeutics, Inc.; Developed

Drug for rare disease

4.

Taltz

ixekizumab

3/22/2016

To treat adults with moderate-to-severe plaque psoriasis.

Eli Lilly and Company

5.

Cinqair

reslizumab

3/23/2016

To treat severe asthma

Teva Respiratory, LLC.

6.

Defitelio

defibrotide sodium

3/30/3016

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

Jazz Pharmaceuticals.

First in class;

Drug for rare disease

7.

Venclexta

Venetoclax

4/11/2016

For chronic lymphocytic leukemia in patients with a specific chromosomal abnormality

AbbVie and Genentech USA Inc.

First in class;

Drug for rare disease

8.

Nuplazid

pimavanserin

4/29/2016

To treat hallucinations and delusions associated with psychosis experienced by some people with Parkinson’s disease

Acadia Pharmaceuticals.

9.

Tecentriq

atezolizumab

5/18/2016

To treat urothelial carcinoma, the most common type of bladder cancer

Genentech USA Inc.

10.

Zinbryta

Daclizumab

5/27/2016

To treat multiple sclerosis

Biogen, Inc.

First in class

11.

Ocaliva

obeticholic acid

5/27/2016

To treat rare, chronic liver disease

Intercept Pharmaceuticals, Inc.

First in class;

Drug for rare disease

12.

Axumin

fluciclovine F 18

5/27/2016

A new diagnostic imaging agent to detect recurrent prostate cancer

Blue Earth Diagnostics, Ltd.,

13.

NETSPOT

gallium Ga 68 dotatate

6/1/2016

A diagnostic imaging agent to detect rare neuroendocrine tumors

Advanced Accelerator Applications USA, Inc.

Drug for rare disease

14.

Epclusa

sofosbuvir and velpatasvir

6/28/2016

To treat all six major forms of hepatitis C virus

Gilead Sciences, Inc

15.

Xiidra

lifitegrast ophthalmic solution

7/11/2016

To treat the signs and symptoms of dry eye disease

Shire US Inc.

First in class

16.

Adlyxin

lixisenatide

7/27/2016

To improve glycemic control (blood sugar levels)

Sanofi-Aventis U.S. LLC

17.

Exondys 51

Eteplirsen

9/19/2016

To treat patients with Duchenne muscular dystrophy

Sarepta Therapeutics.

First in class;

drug for rare disease

18.

Lartruvo

Olaratumab

10/19/2016

To treat adults with certain types of soft tissue sarcoma

Eli Lilly and Company

Drug for rare disease

19.

Zinplava

Bezlotoxumab

10/21/2016

To reduce the recurrence of Clostridium difficile infection in patients aged 18 years or older

Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

First in class

20.

Eucrisa

crisaborole

12/14/2016

To treat mild to moderate eczema (atopic dermatitis) in patients two years of age and older

Anacor Pharmaceuticals, Inc.

21.

Rubraca

rucaparib

12/19/2016

To treat women with a certain type of ovarian cancer

Clovis Oncology, Inc.

Drug for rare disease

22.

Spinraza

Nusinersen

12/23/2016

To treat children and adults with spinal muscular atrophy (SMA)

Marketed by Biogen; Developed by Ionis Pharmaceuticals

First in class; Drug for rare disease

Lower Than Average Approval Number

The number of approved novel drugs in 2016 is less than half of the 45 novel drugs approved in 2015. The chart below illustrates the novel drug filings and approvals in each year of the past decade. The vertical bars in the graph indicate the number of novel drugs approved, and the points connected by lines indicate the number of new applications filed. From 2007 through 2015, CDER received an average of about 36 applications for novel drugs per year, while the average approval number remained about 29 drugs per year. While the approval numbers were down, CDER estimated 41 filings for 2016, higher than the average in recent years.

John Jenkins, director of FDA’s Office of New Drugs, who retired from the FDA on January 7, 2017, provided several explanations for the lower number of novel drugs approved in 2016 on the FDA's blog. One reason he offered was that CDER approved five novel drugs in 2015 that had originally been planned for approval in 2016. Another reason he mentioned was a higher number of Complete Responses (CR) in 2016. Issuance of a CR provides advice on what the sponsor needs to do for FDA to support resubmission of the application. CDER issued 14 CR letters for novel drugs in 2016, higher than in recent years.

Despite the lower number of approvals, The FDA’s Novel Drug Summary for 2016 notes that the approved drugs have the potential to have a big impact in several therapeutic areas. Eight of the drugs approved last year were identified as “first in class” treatments, and nine (41%) of the novel drugs were approved as orphan drugs. An orphan drug is one that treats rare diseases that affect fewer than 200,000 Americans. A majority of the novel drugs approved in 2016 benefited from the FDA's programs to expedite its review process: eight of the twenty-two drugs had a fast track status for drugs having the potential to address unmet needs; seven were considered breakthrough therapies with preliminary clinical evidence demonstrating substantial improvement over other available therapies; fifteen received a priority review designation for review within six months instead of the standard 10 months; and six benefited from the accelerated approval for early approval for a serious or life-threatening illness with benefit over existing therapies. The FDA's report also noted that 19 of the 22 novel drugs were approved in the United States before receiving approval in any other country.