Zevra Therapeutics received a negative CHMP opinion in the EU for arimoclomol in Niemann-Pick disease type C, despite prior U.S. approval. The EMA’s Committee for Medicinal Products for Human Use concluded that efficacy was not sufficiently proven for the new management application. Zevra said it will request a re-examination, and it continues to support eligible patients through an EU expanded access program that has been generating real-world evidence for the drug. The negative opinion is a key commercial and regulatory headwind for the company, given that the therapy is positioned as a disease-modifying complement to miglustat.