The EMA’s Committee for Medicinal Products for Human Use (CHMP) delivered two orphan-drug decisions—backing Minoryx’s ALD therapy while issuing a negative stance on a separate Niemann-Pick disease type C application. In total, eight positive opinions were issued, with Minoryx’s cALD therapy receiving backing after a prior rejection in 2024. Meanwhile, CHMP rejected Zevra’s arimoclomol in NPC, despite the U.S. FDA’s approval of Miplyffa (heat shock protein amplifier, used with miglustat) in September 2024. Zevra said it will request a re-examination after the agency determined efficacy was not sufficiently proven. The mixed CHMP outcomes highlight the EMA’s scrutiny on efficacy thresholds in rare neurodegenerative diseases, and the knock-on impact on development timelines and commercial strategy across global markets.