Zevra Therapeutics Submits MAA to EMA for Arimoclomol (MIPLYFFA®) in Niemann-Pick Disease Type C

29 July 2025 | Tuesday | News

Application aims to bring first disease-modifying therapy for NPC to European patients

Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.

“This EMA submission marks a significant milestone for the Company as we continue to expand access to MIPLYFFA® in NPC patients across the globe,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. “Concurrently, we continue to advance our global Expanded Access Program, with 89 patients enrolled in Europe at the end of Q2, reinforcing MIPLYYFA’s potential to serve as a foundational treatment option across European markets. We extend our deepest gratitude to everyone involved – especially those patients and clinicians who participated in our clinical development programs and look forward to potential European approval of arimoclomol to expand on our approval in the U.S. last year.”

The EMA will review the application under the centralized marketing authorization procedure. If a marketing authorization is granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway.

Adrian Quartel, M.D., Zevra’s Chief Medical Officer added, “The extensive data generated for arimoclomol has shown long-term, meaningful clinical outcomes with 5 to 7 years of patient experience across more than 270 NPC patients through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. We are confident in the strength of our MAA data package and look forward to our interactions with the EMA.”

 

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