Zevra Therapeutics has developed a new drug called acrimoclomol, now FDA-approved to treat Niemann-Pick disease type C (NPC), an ultra-rare metabolic disorder with fatal effects on the central nervous system. NPC is caused by gene mutations affecting cholesterol transport within cells, leading to organ damage and symptoms like mobility loss and cognitive decline. Miplyffa, a capsule taken orally three times a day, has shown to slow disease progression in NPC patients. The drug’s approval also requires combination treatment with miglustat. Zevra acquired the drug from Orphazyme and plans to make Miplyffa available in the U.S. soon, supported by a recent stock offering.
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FDA Approval Makes a Zevra Drug the First Therapy for Rare and Fatal Metabolic Disease
