FDA approves Zevra’s Miplyffa, first treatment for Neimann-Pick disease, type C

The U.S. Food and Drug Administration approved Miplyffa, an oral medication for the treatment of Neimann-Pick disease, type C, or NPC. “Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved by the FDA to treat NPC,” a rare genetic disease that results in progressive neurological symptoms and organ dysfunction, the FDA stated. The FDA granted Miplyffa priority review, orphan drug, rare pediatric disease, fast track and breakthrough therapy designations for this application. The FDA granted approval of Miplyffa to Zevra Therapeutics.