Zevra Therapeutics Announces Final CHMP Opinion on Arimoclomol for the Treatment of Niemann-Pick Disease Type C (NPC) in the European Union
Key Highlights
- ➤Zevra Therapeutics (ZVRA) receives negative CHMP opinion on EU arimoclomol application
- ➤European Commission expected to issue final Marketing Authorization decision within 67 days
- ➤Zevra will continue Expanded Access Program support for eligible arimoclomol patients
- ➤NPC 002 Phase 2/3 trial showed reduced disease progression with arimoclomol plus miglustat
- ➤Long-term open-label data showed arimoclomol treatment effect sustained over multiple years
Expert Statements
Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics
“We are deeply disappointed by the CHMP's opinion, particularly given the significant unmet medical need faced by individuals and families living with NPC”
Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics
“We remain grateful to the patients, caregivers, investigators, advocacy organizations and healthcare providers who have been part of the arimoclomol journey. While this outcome is not what the NPC community hoped for, our priority remains supporting patients and families.”
Sandra Cowie, President of the International Niemann-Pick Disease Alliance (INPDA)
“The NPC community is profoundly disappointed by this decision”
Sandra Cowie, President of the International Niemann-Pick Disease Alliance (INPDA)
“Families affected by NPC face a relentlessly progressive disease that impacts every aspect of daily life. People living with NPC and their caregivers across Europe have been waiting for access to additional treatment options, making this outcome particularly difficult for those who continue to face the daily realities of this devastating disease. INPDA remains committed to advocating for people living with NPC and ensuring that the voices of those living with NPC and their families are heard.”
BOSTON, Nov. 13, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company committed to redefining what's possible in 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 on 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 that affects approximately 1,000 people in Europe. The European Commission is expected to issue its final decision on the Marketing Authorization Application within 67 days.
Zevra will continue to support eligible patients currently receiving arimoclomol through its global Expanded Access Program (EAP), subject to applicable regulatory requirements and in coordination with national authorities. The Company remains focused on helping ensure continuity of treatment for patients currently benefiting from arimoclomol through the program.
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