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Zevra Therapeutics prices rare genetic disorder drug at up to $106,000 per month

By Thomson Reuters Sep 23, 2024 | 7:48 AM

(Reuters) – Zevra Therapeutics’ drug, Miplyffa, for treating a rare and fatal genetic disorder will have a wholesale acquisition cost ranging between $40,000 and $106,000 per month depending on dosage, the company said on a conference call on Monday.

The U.S. health regulator on Friday approved the drug making it the first treatment to get a nod for Neimann-Pick disease type C (NPC) – a rare genetic condition that affects the nervous system and other organs.

The company said the average monthly price is $85,000 per month based on its expanded access program.

H.C. Wainwright analysts estimated average net pricing of the drug to be between $500,000 to $600,000 per year.

Miplyffa, in combination with miglustat, was cleared to treat neurological symptoms associated with NPC in adults and children 2 years of age and older.

(Reporting by Kashish Tandon and Sriparna Roy in Bengaluru; Editing by Shailesh Kuber)