2013 DECIDEDLY AVERAGE FOR THE FDA
The FDA approved 27 new drugs in 2013, down from the 15-year high of 2012 with its 39 approvals. This should not be cause for alarm since 2013 numbers fall in line with the average of 28 over the last five years (FDA.gov).
The FDA attributes the drop to fewer applications received: 32 for 2013 vs. 41 for 2012. Of the 27 approvals, nine were for rare diseases and three were given the newly-established (2012) “breakthrough” designation.
Only two were large molecule drugs: Gazyva, a monoclonal antibody treatment for chronic lymphocytic leukemia marketed by Roche; and Kadcyla, an antibody-drug conjugate for the treatment of breast cancer developed by Genentech. Another approval of note was Kynamro, the first commercially-viable antisense drug approval. Kynamra was developed by ISIS Pharmaceuticals in partnership with Genzyme and is used to treat familial hypercholesterolemia.
BREAKTHROUGH THERAPY DESIGNATION
Breakthrough therapy designation expedites the development and review of drugs for serious or life-threatening conditions. Based on preliminary clinical evidence, the drug may have substantial improvement on at least one clinically significant endpoint over available 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.
BREAKTHROUGH THERAPY DESIGNATION VS. FAST TRACK DESIGNATION
Both designations are intended to expedite the development and review of drugs for serious or life-threatening conditions; however, there are differences in what needs to be demonstrated for each designation. To qualify for breakthrough therapy designation, the drug sponsor must provide preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. In contrast, a drug may qualify for fast track designation by submitting either nonclinical or clinical data that demonstrates the potential to address unmet medical need.