The EMA’s CHMP backed two orphan drug orphan indications while issuing a negative opinion on a therapy already approved in the U.S., underscoring continued divergence between FDA and European evidence thresholds. Among eight positive opinions, Minoryx’s cALD therapy received backing after a prior 2024 rejection. At the same time, CHMP rebuffed Zevra’s arimoclomol application for Niemann-Pick disease type C (NPC), setting up a potential re-examination pathway for the company. The outcomes affect near-term treatment availability in Europe and highlight how quickly companies may need to repackage efficacy and benefit-risk data across jurisdictions for rare disease pipelines.