Zevra Therapeutics has agreed to sell its rare pediatric disease priority review voucher (PRV) for $150 million to an undisclosed buyer.
The PRV was granted to Zevra in September 2024 following the FDA approval of Miplyffa capsules for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.
Miplyffa is the first FDA-approved treatment for the ultra-rare, relentlessly progressive and fatal disease. People living with NPC experience progressive physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation and fine motor skills.
The deal, which is expected to close within 30-45 days, will help fund the commercial launches of Miplyffa capsules and Olpruva, a urea cycle disorders treatment picked up through Zevra’s 2023 acquisition of Acer Therapeutics.