The EMA’s CHMP backed two orphan drugs while rejecting a previously FDA-approved treatment pathway related to Niemann-Pick disease type C, according to regulator updates compiled in BioCentury’s coverage. Minoryx’s cALD therapy cleared the CHMP with a positive opinion after earlier setbacks in 2024. Separately, the CHMP gatekeepers also rebuffed Zevra Therapeutics’ application tied to arimoclomol for Niemann-Pick disease type C. Zevra said it will ask for a re-examination after the committee ruled that efficacy had not been sufficiently proven, despite US approval and an expanded access program generating additional real-world evidence.