FDA Approves Zevra's Miplyffa, First Treatment for Niemann-Pick Disease Type C, Valued at $150 Million

Sep 20, 2024, 03:42 PM

The U.S. Food and Drug Administration (FDA) has approved Zevra Therapeutics' drug, Miplyffa (arimoclomol), for the treatment of Niemann-Pick disease type C (NPC). This approval marks the first treatment for this rare and fatal genetic disorder. The approval also makes Zevra eligible to receive a Priority Review Voucher, valued at approximately $150 million. Miplyffa is approved for use in combination with miglustat for patients aged 2 and older. The New Drug Application (NDA) for arimoclomol had a PDUFA date of September 21, 2024. The US FDA noted that this is the first treatment to get a nod for the condition.