FDA plans to speed path to approval for some gene therapies, starting with hemophilia

The Food and Drug Administration will soon be alerting companies that certain gene therapies in development can qualify for less arduous review at the agency, Commissioner Scott Gottlieb said Tuesday.

Specifically, gene therapies for hemophilia, a rare disease in which blood doesn’t clot properly because it lacks certain proteins, could be evaluated based on whether therapy increases those proteins in the blood, regardless of whether the therapy actually causes the patient to bleed less.

Gottlieb said that hemophilia is the first disease the FDA will target with its new policy. An FDA spokesperson later said hemophilia was chosen because “it’s an area of a lot of development activity.”

Gottlieb made these remarks at the annual board meeting of the Alliance for Regenerative Medicine. It was part of a broad speech about the promise of cell and gene therapies, which he said “will soon become the mainstay of how we treat a wide range of illness.” He said that the agency will release a “comprehensive policy framework” to guide companies on how to evaluate therapies for different sorts of diseases...