EMA Recommends Record Number of Medicines for Rare Diseases

  In 2014, the European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorization 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, according to the agency.

Among these products 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, a rare eye condition that can result in blindness.

Special regulatory pathways were used for these three medicines: conditional marketing authorization for Translarna and Holoclar, and approval under exceptional circumstances for Scenesse. These mechanisms are designed 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 authorization, noted EMA.

Eight new medicines for cancer were recommended for marketing authorization in 2014, of which Lynparza, Imbruvica, Gazyvaro, and Cyramza target rare cancers that are difficult to treat. A targeted treatment for melanoma patients whose cancer has a specific mutation was also recommended for approval in 2014 (Mekinist).

Overall, the number of medicines containing new active substances continues to increase, the agency continued. One in two medicines, either orphan or non-orphan, recommended for approval in 2014, contains a substance that has never been used in medicines before. These medicines have the potential to treat diseases for which no treatments were previously available or bring added benefit to patients over existing therapies.

According to the release issued on January 9, “During the past year, EMA provided more scientific support in the early stages of medicine development. Almost seven out of ten applicants received scientific advice from EMA’s Committee for Medicinal Products for Human Use (CHMP) during the development phase of their medicine and this figure rises to four out of five when it comes to innovative medicines. This is a significant increase compared with 2013 when only half of applicants who had a positive opinion for their medicine had received scientific advice.”

Seven positive opinions were granted after an accelerated assessment in 2014 (Daklinza, Harvoni, Exviera, Viekirax, Ofev, Sylvant, Ketoconazole HRA). Four of these are for the treatment of chronic hepatitis C virus (HCV) infection. The CHMP used the accelerated assessment tool to make a new generation of medicines available to patients potentially more quickly; these treatments have high cure rates and have recently reshaped the way patients with chronic HCV infection can be treated.   Read the release here.