Europe’s EMA CHMP backed Minoryx’s cALD orphan therapy while issuing a negative view for Zevra’s arimoclomol in Niemann-Pick disease type C, according to CHMP recommendations reported in the biotech regulatory track. Minoryx’s Minoryx Therapeutics cleared a key European gate after earlier hurdles, while Zevra will pursue a re-examination after the efficacy concerns. The split highlights how EU orphan programs can diverge sharply from prior FDA outcomes, with Zevra’s therapy having been approved by the US FDA in 2024. Investors and patient advocates will now focus on the next CHMP steps and any additional data Zevra plans to submit for NPC. With the CHMP opinion process now complete for these submissions, the next milestones are national-level decisions across EU member states and whether companies bring further evidence to address endpoints and responder definitions. Overall, the EMA’s posture underscores the regulator’s emphasis on clinical efficacy adequacy in ultra-rare diseases despite expanding access pathways.