Zevra Therapeutics received a priority review voucher for Miplyffa, the first FDA-approved treatment for the rare metabolic disorder Niemann Pick disease type C. Though Zevra has other drug candidates that could benefit from the voucher, the biotech is selling it to an undisclosed buyer.
Zevra Therapeutics drug Miplyffa is now FDA approved for Niemann-Pick disease type C, a lysosomal storage disorder. The affirmative regulatory decision took into account additional clinical data generated since the FDA turned down a submission for the drug in 2021.
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