RegulatoryJul 24, 2026, 07:31 AM
Zevra receives negative CHMP opinion for arimoclomol MAA
AI Summary
Zevra Therapeutics announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol, intended for Niemann-Pick disease type C (NPC). Despite this setback, Zevra plans to request a re-examination of the CHMP opinion and will submit a detailed explanation. The company remains committed to bringing arimoclomol to patients in Europe and continues to provide access through its global Expanded Access Program.
Key Highlights
- CHMP adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol.
- The MAA was for the treatment of Niemann-Pick disease type C (NPC).
- Zevra plans to request a re-examination of the CHMP opinion.
- The company will submit a detailed explanation outlining the basis for the re-examination request.
- Zevra continues to support access to arimoclomol for eligible patients through its global Expanded Access Program.
Price Impact
More from ZVRA