Zevra Therapeutics Inc. (ZVRA) announced that the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA (arimoclomol) capsules as an orally administered treatment for Niemann-Pick disease type C.
This marks the first FDA-approved treatment for Niemann-Pick disease type C, a rare and progressive neurodegenerative disorder.
MIPLYFFA is approved for use in combination with miglustat to address the neurological symptoms of Niemann-Pick disease type C (NPC) in both adult and pediatric patients aged 2 years and older. Additionally, the company disclosed receiving a Rare Pediatric Disease Priority Review Voucher (PRV) alongside the approval.
Zevra Therapeutics has announced the immediate commencement of launch activities for MIPLYFFA, with the treatment expected to be commercially available in the U.S. within eight to twelve weeks.
