CHMP Issues Negative Opinion on Zevra’s NPC Therapy

Rare Daily Staff

European regulators have issued a negative opinion on Zevra Therapeutics’ marketing application for arimoclomol, a setback for the company’s effort to expand access to the rare disease treatment in Europe.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted the opinion on Zevra’s Marketing Authorization Application for arimoclomol for Niemann-Pick disease type C (NPC).

Zevra said it plans to request a re-examination of the decision and will submit a detailed justification as part of that process. “We are committed to investigating appropriate pathways to bring arimoclomol to patients in Europe,” said Zevra CEO Neil McFarlane.

NPC is a rare, progressive, and life-threatening neurodegenerative disorder caused by the body’s inability to properly transport lipids within cells, leading to neurological decline, loss of motor function, and premature death. Treatment options remain limited, underscoring the disease’s significant unmet medical need.

Arimoclomol is a small molecule designed to amplify the body’s cellular stress response by enhancing production of heat shock proteins, which help stabilize misfolded proteins and support cellular function. Zevra said it will continue to provide access through its global Expanded Access Program for eligible patients and gather additional real-world evidence.

The company also noted that updated international clinical management guidelines include arimoclomol as a treatment option for patients with NPC. In the United States, the FDA approved Miplyffa (arimoclomol) in 2024 for use with miglustat to treat neurological manifestations of NPC in adults and children 2 years and older.

Photo: Zevra CEO Neil McFarlane.