Arimoclomol (EAP) - (NPC002)

June 23, 2026: Zevra Therapeutics, Inc. (NASDAQ: ZVRA) announced publication in Molecular Genetics and Metabolism Reports of results from a Phase 2/3 multicenter, open-label pediatric substudy of arimoclomol (MIPLYFFA) plus concomitant miglustat in five infants with Niemann-Pick disease type C (NPC) aged 12 to <24 months at enrollment, evaluating safety, tolerability, and pharmacokinetics over up to 36 months. Arimoclomol was generally well tolerated with no new safety signals, and pharmacokinetics were consistent with older pediatric populations; developmental assessments showed variable delays consistent with underlying disease heterogeneity. The company said the small study provides preliminary evidence supporting initiation of MIPLYFFA in this age group and supports exploration of earlier treatment.

Regulatory context noted in the release: MIPLYFFA is FDA-approved (Sept. 20, 2024) for use with miglustat to treat neurological manifestations of NPC in adults and pediatric patients aged ≥2 years, and Zevra has submitted a Marketing Authorization Application to the EMA for arimoclomol in NPC.