Zevra Therapeutics Announces Multiple Datasets on MIPLYFFA® (arimoclomol) to be Presented at the International Congress of Inborn Errors of Metabolism (ICIEM)

CELEBRATION, Fla., Aug. 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced four posters on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM), taking place September 2-6, 2025, in Kyoto, Japan.

MIPLYFFA is approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC). A poster (# BP-19) detailing that MIPLYFFA’s mechanism of action uniquely targets the underlying pathophysiology of NPC was selected for a Best Poster award. Additionally, positive, new data from a multi-center pediatric substudy in patients younger than two years old, as well as a new prespecified efficacy analysis of patients on routine clinical care with miglustat who switched from placebo to MIPLYFFA in their treatment regimen, will be presented.

Poster Details