The European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorization in a single year in 2014. Out of the 82 medicines for human use recommended last year, 17 are meant for the treatment of a rare disease, serving patients who frequently have a few or no treatment options.
Among them is the first medicine for the treatment of Duchenne muscular dystrophy (Translarna) as well as the first treatment for erythropoietic protoporphyria, a rare genetic disease called Scenesse that makes exposure to light intolerable.
The first recommendation worldwide of a therapy-based on stem cells also arrived in 2014. The orphan medicine (Holoclar) is a treatment for limbal stem cell deficiency (LSCD), a rare eye condition that can result in blindness.