Zevra Publishes Phase 2/3 Data: MIPLYFFA Well Tolerated in Five NPC Infants Over 36 Months
ZVRA•Zevra Therapeutics reported phase 2/3 substudy results for five infants (12 to <24 months) with Niemann-Pick disease type C showing MIPLYFFA (arimoclomol) was well tolerated over up to 36 months with no new safety signals. Pharmacokinetics aligned with older pediatric patients and developmental assessments revealed variable delays, supporting earlier treatment.
1. Study Design and Patient Population
The open-label, multicenter phase 2/3 substudy evaluated MIPLYFFA (arimoclomol) plus miglustat in five infants aged 12 to under 24 months diagnosed with Niemann-Pick disease type C. Patients received treatment for up to 36 months, with safety, tolerability and pharmacokinetics assessed at scheduled intervals throughout the study.
2. Safety and Tolerability Results
Over the treatment period, MIPLYFFA was generally well tolerated in all five infants. No new safety signals emerged, and adverse events aligned with the known profile of arimoclomol. Hypersensitivity reactions and creatinine increases were monitored, with no severe events prompting discontinuation in this cohort.
3. Pharmacokinetic and Developmental Findings
Pharmacokinetic parameters in infants matched those observed in older pediatric studies, indicating consistent drug exposure. Developmental assessments revealed variable delays reflective of underlying phenotypic heterogeneity. These preliminary findings support exploration of earlier MIPLYFFA initiation to potentially improve long-term outcomes in this age group.
