Business
Zevra Therapeutics Presents Positive New Real-World Data on MIPLYFFA® in Patients with Neimann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium™
Real-world and clinical trial data underscore long-term safety and effectiveness in pediatric and adult NPC patientsCELEBRATION, Fla., Feb. 04, 2026 (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 the presentation of four posters highlighting positive new data on MIPLYFFA® (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the
About this update from Zevra Therapeutics, Inc.
[{"type":"image","alt":"Zevra Therapeutics","displaySize":"","headline":null,"caption":"Zevra Therapeutics","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":80,"url":"https://media.zenfs.com/en/globenewswire.com/47aa28c5ce5943ac0fb423dcc40d01c5"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/BTrlkcW3K5pHsBf7HqZtVg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTExMjtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/47aa28c5ce5943ac0fb423dcc40d01c5","width":300,"height":80}},"lazy":false},{"type":"text","content":"Real-world and clinical trial data underscore long-term safety and effectiveness in pediatric and adult NPC patients","length":116,"tagName":"p"},{"type":"text","content":"CELEBRATION, Fla., Feb. 04, 2026 (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 the presentation of four posters highlighting positive new data on MIPLYFFA® (arimoclomol) for the treatment of Niemann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium™.","length":412,"tagName":"p"},{"type":"text","content":"“These data highlight MIPLYFFA’s potential to meaningfully stabilize disease progression across a broad spectrum of NPC patients, including adults who have historically had limited clinical data,” said Adrian Quartel, M.D., FFPM, Zevra’s Chief Medical Officer. “As controlled clinical trial results are reinforced by long-term real-world experience, we continue to strengthen our understanding of MIPLYFFA’s impact and advance our mission to deliver meaningful therapies for people living with rare diseases.”","length":509,"tagName":"p"},{"type":"text","content":"Key Data Highlights","length":19,"tagName":"p"},{"type":"list","items":[{"val":[{"type":"text","content":"“Real-world Safety and Effectiveness of Arimoclomol in Patients with NPC: Outcomes from the U.S. Early Access Program (EAP) Over a 4-Year Period” (Podium Presentation/Poster 41)","length":177,"tagName":"p"},{"type":"list","items":[{"val":[{"type":"text","content":"Four years of real-world data from the U.S. EAP demonstrate that arimoclomol was well tolerated and stabilized disease progression in the overall cohort, with changes in clinical severity scores remaining below thresholds for clinically meaningful worsening, supportin...