Zevra Therapeutics Announces Publication of Four-Year Real-World Data from U.S. Early Access Program Evaluating MIPLYFFA® (arimoclomol) in Niemann-Pick Disease Type C (NPC)

BOSTON, Aug. 03, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today announced the publication of “Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US Early Access Program over a 4-year period” in Molecular Genetics and Metabolism.

The longitudinal study evaluated arimoclomol in a broad US NPC population spanning a wide range of ages and disease severity, generating up to four years of follow-up data from routine clinical practice. The study includes the first published real-world data evaluating arimoclomol in adults with NPC, a historically understudied population.