WASHINGTON — 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.”
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