With the approval of Spinraza (nusinersen) by Biogen in late December, the books close on FDA approvals of new molecular entities (NMEs) in 2016. The total for the year is surprisingly low: 20, well below the 45 approved in 2015, and a level not seen since the mid-2000s, which is looked back on as a painful lull in new drug entries for the industry. Spinraza is an orphan, pediatric drug to treat a genetic disorder, spinal muscular atrophy, and is the eighth orphan drug in FDA’s list of 20.
The annual CDER New Drug Review, issued mid-month by Dr. John Jenkins, director of the Office of New Drugs, attributes some of the decline to accelerated approvals granted in 2015 that could easily have fallen into 2016, a higher number of complete response letters (which essentially kill or cause a rewrite of a drug application), along with a lesser number of “NME actions” during the year. The NME actions point to a lessening in manufacturers’ readiness to file an application in the first place; but that decline was only from 40 in 2015 to 36 as of early December. (Applications have only a rough relationship to approvals, since FDA reviews are not usually completed in the calendar year of submission.)
Although nearly half (8 of 20) of the approvals are for rare diseases, which affect a few thousands to 200,000 patients and therefore have a limited market, analysts and press reports point to some potential blockbusters once the drugs are on the market; these include Genentech’s Tecentriq (atezolizumab) for lung cancer, Merck’s Zepatier (grazoprevir/elbasvir) for hep C, and Lilly’s Taltz (ixekizumab) for plaque psoriasis. And, although the workings of the recently passed 21st Century Cures Act remain up in the air until FDA regulations come out, that law’s loosening of evidence standards for approval, and expanded attention to regenerative medicine technologies, is expected to boost new drug development.
Meanwhile, ever mindful of the politics embedded in FDA policy, the Jenkins report also notes that his agency still dominates the global market in terms of first approvals, with the US responsible for 65% of the new drugs approved in 2015 (a new high), while no other region of the world tops 20%. The report also documents that the time to approval (of, of course, approved drugs) has risen steadily with each new PDUFA (Prescription Drug User Fee Act, which is renewed every five years), going from about 15% approvals within 12 months pre-PDUFA, to about 75% within 12 months for the current PDUFA cohort.