In 2014, the European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorisation in a year. Out of the 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.

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 which causes intolerance to light (Scenesse).

The past year also saw the first recommendation worldwide of a therapy based on stem cells. The orphan medicine (Holoclar) is a treatment for limbal stem cell deficiency (LSCD), a rare eye condition that can result in blindness.

Special regulatory pathways were used for these three medicines (conditional marketing authorisation for Translarna and Holoclar, and approval under exceptional circumstances for Scenesse). These mechanisms are in place to potentially speed up market access for medicines that fulfill unmet medical needs but for which comprehensive data cannot be provided at the time of application for a marketing authorisation.

For more details, go to: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2015/01/news_detail_002247.jsp&mid=WC0b01ac058004d5c1