Zevra Therapeutics Announces Open Label Extension Data Showing Sustained Long-Term Efficacy of MIPLYFFA for the Treatment of NPC Published in the Journal of Molecular Genetics and Metabolism

CELEBRATION, Fla., July 16, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced the publication of “Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial” in the peer-reviewed journal, Molecular Genetics and Metabolism (https://doi.org/10.1016/j.ymgme.2025.109189). MIPLYFFA^® (arimoclomol) is an approved treatment for Niemann-Pick disease type C (NPC), a neurodegenerative disease caused by lysosomal dysfunction.

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