EMA’s CHMP backed two orphan drug therapies and rejected a separate option tied to an FDA-approved NPC treatment. In the BIOCentury regulation coverage, Minoryx’s cALD therapy received backing after a 2024 rejection, while the CHMP opinion rebuffed an FDA-approved NPC therapy. The report frames the decision as a renewed European assessment gap versus the US label, underscoring how orphan indications can diverge across regulators when evidence packages and endpoint interpretations differ. For sponsors targeting cross-region adoption, the outcome reiterates the need to align clinical evidence strategy early for both EMA and FDA pathways, especially where the evidentiary threshold for efficacy differs between committees.