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How many countries will approve Miplyffa for Niemann-Pick disease type C treatment by Dec 31, 2024?
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Resolution / Starting Odds
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Official announcements from regulatory agencies and Zevra Therapeutics
Story
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.
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