2 June 2026 - The US FDA is more likely to approve drugs with an orphan designation that were indicated for subgroups of cancer or paediatric indications than the EMA, the results of a recent cross-sectional study suggest.

Enrico Costa, of the Italian Medicines Agency (AIFA) and the Program On Regulation, Therapeutics, And Law (PORTAL) at Brigham and Women’s Hospital and Harvard Medical School, and colleagues said FDA licensed nearly all approved products with an orphan drug designation between 2011 and 2020. The FDA and EMA both approved more than one-third of the total orphan drugs in both regions, about one-third of orphan drugs were approved by FDA only, and less than one-third of FDA approved drugs with an orphan drug designation were approved in the European Union without that designation, they noted.

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