Publication of Positive MIPLYFFA Pediatric Substudy Data in Infants with Niemann-Pick Disease Type C – Zevra Therapeutics

 Zevra Therapeutics announced the publication of “Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy” in Molecular Genetics and Metabolism Reports. This multicenter, open-label pediatric substudy evaluated arimoclomol, in addition to concomitant miglustat, in five children with Niemann-Pick disease type C (NPC) aged 12 to <24 months at enrollment, assessing safety, tolerability, and pharmacokinetics over up to 36 months of treatment.

“The publication of these data adds to the growing body of clinical evidence supporting MIPLYFFA across age groups in NPC. Understanding the safety and pharmacokinetic profile of arimoclomol in these young children is critical as we continue to explore the potential role of earlier treatment initiation for patients living with this devastating disease.” – Christine i Dali, Zevra’s Vice President, Clinical Science and an author of the publication.

Arimoclomol was generally well tolerated, with no new safety signals, and pharmacokinetic results were consistent with older pediatric populations. Developmental assessments showed variable delays, reflecting underlying phenotypic heterogeneity. Although limited by small sample size, the study provides preliminary evidence supporting initiation of MIPLYFFA in this age group.