BOSTON, Mass. — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease type C (NPC), a rare, progressive and life-threatening neurodegenerative disorder.
Under European regulatory procedures, the Company plans to request a re-examination of the CHMP opinion and submit a detailed explanation outlining the basis for the request.
“While the review did not result in the outcome we had hoped for, we are committed to investigating appropriate pathways to bring arimoclomol to patients in Europe,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics. “We appreciate the important role the CHMP plays in assessing therapies across Europe, and we remain confident in the totality of evidence supporting the potential of arimoclomol to make a meaningful difference in the lives of patients living with NPC.”
Zevra remains steadfast in its commitment to the NPC community and believes that patients facing this devastating disease deserve access to potential treatment options. Through its global Expanded Access Program (EAP), the Company continues to support eligible patients to advance access to arimoclomol and generate additional real-world evidence of its clinical impact. The recently updated international Consensus Clinical Management Guidelines for Niemann-Pick disease type C further underscore the role of arimoclomol as an important treatment option for individuals living with NPC.1
“In my experience, arimoclomol has demonstrated meaningful treatment effects in NPC, with stabilization and slowed progression in a condition with significant unmet need,” said Prof. Thorsten Marquardt, University of Münster. “Real-world clinical experience in NPC can provide additional insights into how patients are managed in routine care settings, contributing to the broader understanding of disease management in ultra-rare, progressive diseases.”
About Zevra Therapeutics, Inc.
Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra’s vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value.
For more information, please visit www.zevra.com or follow us on X and LinkedIn.
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Reference
- T. Hiwot, F. D. Porter, T. Bremova-Ertl, et al., “2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C,” Journal of Inherited Metabolic Disease 49, no. 3 (2026): e70185, https://doi.org/10.1002/jimd.70185.