Zevra Therapeutics to Present New Long-Term Real-World Data on MIPLYFFA® in Niemann-Pick Disease Type C (NPC) at the 2026 SSIEM Annual Symposium
Zevra Therapeutics announced the presentation of long-term real-world data on MIPLYFFA (arimoclomol) for treating Niemann-Pick disease type C at the 2026 SSIEM Annual Symposium. Data from U.S. and German early access programs demonstrate sustained disease stabilization over up to 8 years with a consistent safety profile across diverse patient populations, including adults and children. The pivotal trial showed a 65% reduction in disease progression compared to placebo.