Zevra Publishes Positive Phase 2/3 Pediatric Substudy Results for MIPLYFFA in NPC
Zevra Therapeutics announced the publication of results from its Phase 2/3 open-label pediatric substudy of arimoclomol (MIPLYFFA) in infants with Niemann-Pick disease type C (NPC). The study, published in Molecular Genetics and Metabolism Reports, evaluated arimoclomol in five children aged 12 to <24 months, demonstrating that the drug was generally well tolerated with no new safety signals. Pharmacokinetic results were consistent with older pediatric populations, providing preliminary evidence to support earlier treatment initiation in this age group. MIPLYFFA was approved by the U.S. FDA on September 20, 2024, and Zevra has also submitted a Marketing Authorization Application to the European Medicines Agency.
Key Highlights
- Zevra Therapeutics announced publication of Phase 2/3 pediatric substudy results for arimoclomol (MIPLYFFA) in Niemann-Pick disease type C (NPC).
- The open-label study evaluated arimoclomol in five children with NPC aged 12 to <24 months at enrollment.
- The study assessed safety, tolerability, and pharmacokinetics over up to 36 months of treatment.
- Arimoclomol was generally well tolerated with no new safety signals observed.
- Pharmacokinetic results were consistent with those observed in older pediatric populations.
- The study provides preliminary evidence supporting initiation of MIPLYFFA in this young age group.
- MIPLYFFA was approved by the U.S. FDA on September 20, 2024, for NPC treatment.
- Zevra has submitted a Marketing Authorization Application to the EMA for arimoclomol.
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