EMA’s CHMP backed two orphan drugs while issuing a negative stance on a therapy already approved in the US, underscoring how evidence standards are being applied differently across regulators. Minoryx’s cALD program received positive momentum after a prior 2024 rejection, with the committee supporting an orphan-pathway route. At the same time, CHMP rebuffed the FDA-approved NPC therapy, closing off that specific option in the EU for now. The outcome sends companies back to the evidence and protocol design that European reviewers want to see for efficacy in rare neurodegenerative disease settings.