Is research into orphan diseases on the rise?
Stephanie Sutton |
Judging from recent statistics from the European Medicines Agency (EMA), perhaps so. A record number of positive opinions for medicines for rare diseases were issued by the EMA in 2014: 17 positive opinions (compared with just 11 in 2013), including the first medicine for Duchene muscular dystrophy (Translarna; PTC Therapeutics) and the first treatment for erythropoietic protoporphyria (Scenesse; Clinuvel). 2014 also saw another first, with the EMA recommending a therapy based on stem cells. Eight new medicines for cancer were also recommended. Overall, the EMA recommended 82 medicines for approval in 2014.
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