The FDA released its report titled “Novel New Drugs 2016 Summary,” in which they discuss 2016 FDA new drug approvals. In 2016, the FDA’s Center for Drug Evaluation and Research (CDER) approved 22 novel new medicines. The number of approvals in 2016 was down from 2015 with 45 approvals and down from 2014 with 41 approvals. In fact, in looking at approvals over the past five years, 2016 had the lowest number of approvals overall, however the number of new drug filings remained consistent.

Other highlights from the report include:

• 8 of the 22 novel drugs were considered “first in class” which means that they utilize a novel or unique mechanism of action over existing therapies.

• 9 of the 22 NMEs were approved to treat orphan diseases. Orphan diseases are considered rare diseases that affect 200,000 or fewer Americans.

• 16 of the 22 NMEs were designated in one or more expedited pathway categories – Fast Track, Breakthrough, Priority Review, and Accelerated Approval

Fast Track designation is identified by FDA as drugs with the potential to address unmet medical needs. “Fast Track speeds new drug development and review, for instance, by increasing the level of communication FDA allocates to developers and by enabling developers to use a “rolling review” process such that CDER can review portions of an application ahead of the submission of the full application.” (8 novel drugs had this designation)

Breakthrough designation is identified by FDA as drugs with preliminary clinical evidence that shows the potential of substantial improvement over at least one clinically significant endpoint compared with current therapy. “A breakthrough therapy designation conveys all of the fast track program features as well as more intensive FDA guidance on an efficient drug development program.” (7 novel drugs had this designation)

Priority Review is determined by FDA that the drug has the potential to provide a significant advance on medical care. With priority review, FDA sets a target to review the drug within six months instead of the standard that is 10 months. (15 novel drugs had this designation)

Accelerated Approval allows early approval of a drug for a serious or life-threatening illness that offers benefits over current treatments. “This approval is based on a “surrogate endpoint” (e.g., a laboratory measure) or other clinical measure that FDA considers reasonable likely to predict clinical benefit. After this approval, the drug must undergo additional testing to confirm that benefit; this speeds the availability of the drug.” (6 novel drugs had this designation)

• 21 of the 22 were approved on the first cycle, which means that they were approved without the need for additional information that would delay approval.

• 19 of the 22 were approved first in the United States before any other country.

Novel Biologic Drugs Approved by CDER in 2016

Included in the report were 7 biotech drugs or biologics that were approved by CDER. This number was down from last year where there were 13 approved. In addition, 5 of the 7 approved biologics participated in at least one expedited pathway designation.

For the previous year’s coverage, please see:

• 2015 FDA New Drug Approvals – 13 Biologics Receive Approval
• 2014 FDA New Drug Approvals – 11 Biologics Receive Approval
• 2013 FDA New Drug Approvals