Zevra Therapeutics Publishes Arimoclomol Study Results in Niemann-Pick Disease Children
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 aged 12 to less than 24 months at enrollment, assessing safety, tolerability, and pharmacokinetics over up to 36 months of treatment. 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.
