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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.


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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