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.
Rare Record
Is research into orphan diseases on the rise?