FDA Commissioner Plans to Create a Fast Track Approval Pathway for Certain Gene Therapies

During a speech on May 22, 2018 at the Alliance for Regenerative Medicine’s Annual Board Meeting, Food and Drug Administration (FDA) Commissioner Scott Gottlieb announced that the FDA plans to alert companies that “certain gene therapy products” will soon be able to qualify for fast track approval.[1]  The Commissioner explained that the first therapeutic area the FDA will focus on for fast track approval is hemophilia.  He did not say what other diseases may be eligible in the future.  According to an FDA spokesperson, hemophilia was chosen as the first therapeutic area because “it’s an area of a lot of development activity.”[2]  Furthermore, it was announced that the “FDA intends to release a suite of draft guidance documents articulating its framework for the manufacturing and clinical development of gene therapy products,” which will include guidance on product-related issues as well as recommendations for clinical development in certain therapeutic areas.

In contrast to traditional small molecule medicines and biologics, gene therapies treat diseases by changing an individual’s genetic makeup.  Aware of the distinct advantages of gene therapies, Commissioner Gottlieb also identified a number of challenges to bringing gene therapies to market.  These challenges include unique manufacturing issues, product scalability issues, and a relatively unknown long term effectiveness of gene therapy treatments.  Despite the challenges, Commissioner Gottlieb believes that these “are the sort of questions that [the FDA] can help resolve by working together” with gene therapy companies.

It is interesting to note that not long after the Commissioner’s announcement, the FDA put Crispr Therapeutics and Vertex Pharaceuticals’ planned trial of their sickle-cell disease gene editing therapy to on hold.[3]  Although the FDA instituted the hold so that they could request additional information, the FDA has not yet indicated what additional information they require or if this gene therapy could qualify for fast track approval in the future.[4]