Zevra to Seek Re-examination of EMA’s Negative Ruling on Arimoclomol for Niemann-Pick Disease Type C

Zevra Therapeutics (ZVRA) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the company’s Marketing Authorization Application for Arimoclomol to treat Niemann-Pick disease type C.

Niemann-Pick disease type C (NPC) is a rare, progressive, and life-threatening neurodegenerative disorder.

Under European regulatory procedures, Zevra plans to request a re-examination of the CHMP’s negative opinion and submit a detailed explanation outlining the basis for the request. CEO Neil F. McFarlane stated the outcome was disappointing, but the company remains committed to exploring pathways to bring Arimoclomol to patients in Europe, emphasizing confidence in the totality of evidence supporting the drug’s potential to make a meaningful difference for individuals living with NPC.

Zevra continues to support access to Arimoclomol through its global Expanded Access Program, which provides treatment to eligible patients and generates real-world evidence of its clinical impact. Updated international consensus guidelines for NPC further underscore the role of Arimoclomol as an important treatment option.

Prof. Thorsten Marquardt of the University of Münster noted that clinical experience with Arimoclomol has demonstrated meaningful treatment effects in NPC, including stabilization and slowed disease progression in a condition with significant unmet need.

Zevra reaffirmed its commitment to the NPC community, highlighting its broader strategy of advancing therapies for rare diseases and expanding access globally.

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