Zevra Therapeutics To Highlight 4-Yr Real-World Safety And Efficacy Data Of MIPLYFFA In Patients With Niemann-Pick Disease Type C During Podium Presentation At 22nd Annual WORLDSymposium

Four abstracts highlighting clinical data and real-world experience with MIPLYFFA accepted for presentation, including an oral presentation by Caroline Hastings, MD

Company to host satellite symposium on diagnostic and therapeutic advances in Niemann-Pick disease type C (NPC)

CELEBRATION, Fla., Jan. 26, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NASDAQ:ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that four abstracts discussing data and clinical experience associated with MIPLYFFA® (arimoclomol) have been accepted for presentation at the 22nd Annual WORLDSymposium™. Dr. Caroline Hastings will present four-year real-world safety and efficacy data of MIPLYFFA in patients with Niemann-Pick Disease Type C (NPC) during a podium presentation.

The poster presentations include outcomes from the U.S. Early Access Program (EAP), multi-year subgroup analyses, and efficacy results from a randomized, placebo-controlled clinical trial, further expanding the body of evidence characterizing MIPLYFFA in pediatric and adult NPC patients. In addition, Zevra will host a non-continuing education (Non-CE) sponsored satellite symposium focused on diagnostic and therapeutic challenges in NPC.