The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions for two orphan drugs, while rejecting an application involving an FDA-approved NPC therapy. In the orphan-drug review package, Minoryx’s cALD program gained backing after a prior 2024 rejection, according to BioCentury’s regulation coverage. At the same time, CHMP’s decision on Niemann-Pick disease type C (NPC) reinforced a difficult evidence bar in Europe even after a US approval—pushing sponsors toward re-examination pathways or new evidence generation.