Zevra Therapeutics Announces Publication of Four-Year Real-World Data from U.S. Early Access Program Evaluating MIPLYFFA® (arimoclomol) in Niemann-Pick Disease Type C (NPC)
Zevra Therapeutics announced the publication of four-year real-world data in Molecular Genetics and Metabolism demonstrating that MIPLYFFA maintained stable disease severity scores in patients with Niemann-Pick disease type C (NPC). The US Early Access Program study evaluated 109 participants across 14 sites, with mean arimoclomol exposure of 820 days, showing safety observations consistent with the established profile of the therapy.
Key figures
- Drug Name
- MIPLYFFA (arimoclomol)
- Sites
- 14
- Participants
- 109
- Adult participants
- 53
- Mean exposure days
- 820
- Study duration years
- 4
- Concomitant miglustat patients
- 71
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