FDA approves Miplyffa for treatment of Niemann-Pick disease, type C

The U.S. Food and Drug Administration has approved Zevra Therapeutics’ Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC).

Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children aged 2 years and older. The approval received priority review, orphan drug, rare pediatric disease, fast track, and breakthrough therapy designations.

The approval is based on results from a randomized, double-blind, placebo-controlled 12-month trial in patients (ages 2 to 19 years) with a molecularly confirmed diagnosis of NPC.

The analysis included 50 patients randomly assigned (2:1) to treatment with weight-adjusted Miplyffa (31 to 124 mg) or placebo orally three times per day. Miplyffa resulted in slower disease progression as measured by the rescored four-domain NPC Clinical Severity Scale score. The most common side effects of Miplyffa treatment included upper respiratory tract infection, diarrhea, and decreased weight.

“The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering,” Janet Maynard, M.D., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA Center for Drug Evaluation and Research, said in a statement.

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