Zevra Therapeutics said it will pursue a re-examination after CHMP adopted a negative opinion on arimoclomol for Niemann-Pick disease type C. The company’s submission for a marketing authorization application was rejected on grounds that efficacy had not been sufficiently proven. The CHMP outcome follows the FDA’s September 2024 greenlight for Miplyffa, which uses arimoclomol in combination with miglustat, and Zevra’s ongoing expanded access program aimed at generating additional real-world evidence. For Zevra and other rare-disease sponsors, the decision highlights how EMA pathway outcomes can differ from FDA approvals and can materially affect time-to-access and pricing strategy across markets.