FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C, $150 Million Voucher

Sep 20, 2024, 01:40 PM

The U.S. Food and Drug Administration (FDA) has approved Zevra Therapeutics' drug, arimoclomol, also known as Miplyffa, for the treatment of Niemann-Pick disease type C (NPC), a rare and fatal genetic disorder. This approval marks the first oral medication to receive FDA approval for NPC. In addition, Zevra Therapeutics is now eligible to receive a Priority Review Voucher worth approximately $150 million. The approval was announced on September 20, 2024, ahead of the NDA PDUFA date of September 21, 2024.