The FDA’s Breakthrough Therapy Designation — created when the FDA Safety and Innovation Act was signed on 9 July 2012 — is in its second year after a successful start.  Enacted to provide another mechanism to accelerate the path of critical therapeutics to market, the statute has exceeded expectations.  The regulatory agency has granted the designation a total of 47 times as of April 30, 2014, compared to initial estimates that only 3 or 4 drugs per year would earn the title — and the advantages that follow.  To date, 7 approvals have already been granted including Gilead’s controversial runaway blockbuster Sovaldi.

So what does the FDA consider a “breakthrough”?  These therapies must both treat a serious or life threatening condition and demonstrate a substantial improvement over one or more clinical endpoints.  The program is intended for early-stage development… as early as Phase 1, and ideally no later than Phase 2.  For the designees, it will mean an early and intense commitment from the FDA to provide guidance on drug development, as well as eligibility for all the benefits of the FDA Fast Track program, such as Accelerated Approval and Priority Review.