FDA Approves Zevra's Oral Drug Miplyffa for Niemann-Pick Disease Type C

Sep 20, 2024, 05:11 PM

The U.S. Food and Drug Administration (FDA) has approved Zevra Therapeutics' drug, Miplyffa, as the first oral medication to treat Niemann-Pick disease, type C (NPC). This approval, announced on September 20, 2024, marks a significant milestone for the treatment of this rare and fatal genetic disorder, which affects neurons. Parents and advocates have pushed for years for treatments for NPC, facing numerous setbacks before achieving this breakthrough.