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Patients with a rare disease are asking the Food and Drug Administration a question the agency rarely hears: Is that really your final answer?

A group for people with familial chylomicronemia syndrome (FCS), a genetic condition that causes fat to build up in a person’s blood, is circulating a petition that begs the FDA to reconsider its decision to reject the one experimental treatment for FCS, Akcea’s volanesorsen. As of Monday, the petition had more than 8,000 signatures.

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Right now, their quest is still something of a long shot. But if patient advocates and drug companies have anything to say about it, the FDA will hear a version of their question more and more often. Increasingly, patients and the drug companies they often work with are hoping the agency will give more weight to the patient perspective — as some suggest it did when it approved Sarepta’s Exondys 51, a drug for Duchenne muscular dystrophy.

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