BOSTON – July 24, 2026
Zevra Therapeutics, Inc. has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on the Marketing Authorization Application (MAA) for arimoclomol, an investigational treatment for Niemann-Pick disease type C (NPC), a rare, progressive, and life-threatening neurodegenerative disorder. Despite the setback, the company confirmed it will request a formal re-examination of the CHMP decision in accordance with European regulatory procedures and submit a detailed scientific justification supporting its application. Zevra stated that it remains committed to bringing arimoclomol to patients across Europe while continuing to generate additional clinical and real-world evidence supporting the therapy’s potential benefits for individuals living with this ultra-rare disease.
Company Plans Regulatory Re-Examination Following CHMP Decision
Following the negative CHMP opinion, Zevra Therapeutics will initiate the European regulatory re-examination process, allowing the company to provide additional explanations and supporting evidence for arimoclomol’s benefit-risk profile. President and Chief Executive Officer Neil F. McFarlane acknowledged the outcome was disappointing but emphasized the company’s commitment to pursuing every appropriate regulatory pathway to make the therapy available in Europe. Zevra expressed confidence in the totality of clinical evidence supporting arimoclomol, stating that the available data continue to demonstrate its potential to make a meaningful difference for patients affected by Niemann-Pick disease type C, a condition with significant unmet medical need and limited treatment options.
Expanded Access Program Continues to Support Eligible Patients
While the regulatory review continues, Zevra Therapeutics confirmed that eligible patients will continue to receive arimoclomol through the company’s global Expanded Access Program (EAP). The program provides treatment access while also contributing valuable real-world clinical evidence regarding the therapy’s long-term impact. The company noted that recently updated international Consensus Clinical Management Guidelines for Niemann-Pick disease type C recognize arimoclomol as an important treatment option for patients living with the disease. Professor Thorsten Marquardt of the University of Münster highlighted that clinical experience with arimoclomol has demonstrated stabilization and slower disease progression in patients with NPC, while emphasizing the importance of real-world evidence in improving the understanding and management of ultra-rare neurodegenerative disorders.
Advertisement
Rare Disease Strategy Remains a Core Focus for Zevra
Zevra Therapeutics continues to strengthen its position as a commercial-stage rare disease biotechnology company focused on developing and expanding access to therapies for patients with serious genetic disorders. The company stated that advancing arimoclomol remains a strategic priority alongside broader efforts to expand its commercial presence, progress its late-stage pipeline, and achieve additional regulatory milestones. By combining ongoing clinical development with expanded patient access initiatives and real-world evidence generation, Zevra Therapeutics aims to support future regulatory decisions while continuing its mission of delivering innovative treatments to individuals living with rare, progressive diseases worldwide.
Source: Zevra Therapeutics press release
