Zevra Therapeutics (NASDAQ: ZVRA) has announced the publication of data suggesting that MIPLYFFA (arimoclomol) was well tolerated in pediatric patients with Niemann-Pick disease type C (NPC) in the peer-reviewed journal Molecular Genetics and Metabolism Reports.
The 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.
In a statement, Christine i Dali, MD, Vice President, Clinical Science at Zevra and an author of the publication, commented, “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.”
