A European Medicines Agency committee has recommended against approving Zevra Therapeutics’ arimoclomol for Niemann-Pick disease type C, a rare neurodegenerative disorder. The European Commission is expected to make the final decision on the marketing application within 67 days, Zevra said Friday.
The negative opinion came from the EMA’s Committee for Medicinal Products for Human Use, which assesses medicines’ quality, safety and efficacy and advises the European Commission on whether to approve them.
Zevra said it believes evidence submitted for arimoclomol, including results from a Phase 2/3 trial, supports the drug’s therapeutic benefit in NPC. The company said it will continue providing arimoclomol through its global expanded access program to eligible patients who are already receiving it, and will continue work to advance scientific understanding of the disease.
“We are deeply disappointed by the CHMP’s opinion, particularly given the significant unmet medical need faced by individuals and families living with NPC,” Chief Executive Neil McFarlane said.
NPC affects about 1,000 people in Europe. Zevra shares fell 6.0% to $10.49 in premarket trading Friday.
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