Hemophilia gene therapies put on fast track to FDA approval

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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 [May 22].

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.”


One challenge, Gottlieb said, is that the traditional clinical trials companies need to complete before bringing a drug to the market may not be able to fully determine the “durability of response.” In other words, the effectiveness of the therapy might change after the trial is over, and initial signs that the drug works might not pan out upon further scrutiny. But because some of these treatments address patients with few other options, it may be worth the risk for them to undergo a treatment with indirect evidence of a benefit — protein levels in the case of hemophilia — even if there are some unknown future risks.

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Read full, original post: FDA plans to speed path to approval for some gene therapies, starting with hemophilia

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