EMA backs record number of drugs for rare diseases in 2014

The European Medicines Agency recommended the highest number of orphan designated medicines for marketing authorization in 2014. Out of the total 82 medicines for human use recommended in 2014, 17 are intended for the treatment of a rare disease, providing therapies for patients who often have only few or no treatment options.

This just beats the total of 15 approvals for medicine designated as orphan drugs (ie, for rare diseases) that were approved by the US Food and Drug Administration, out of its total 41 approvals in 2014. The EMA total approvals figure includes generic medicines, which the FDA total does not.

Among them is the first medicine for the treatment of Duchenne muscular dystrophy – Translarna (ataluren) from PTC Therapeutics, as well as the first treatment for erythropoietic protoporphyria, a rare genetic disease which causes intolerance to light – Scenesse (afamelanotide), from Australian drugmaker Clinuvel.