2014 was a good year for new orphan drugs in Europe, with a record number put forward for approval by the European Medicines Agency’s Committee for Medicinal Products for Human Use, as the overall total inched up from last year.

Eighty-two human medicines were recommended for marketing clearance in 2014, 17* of which were intended for the treatment of a rare disease. Last year, 81 new medicines got the CHMP nod, 11 of which were orphan drugs.

Of particular note in 2014, the CHMP endorsed the first medicine for Duchenne muscular dystrophy (Translarna), the first treatment for the rare genetic disease erythropoietic protoporphyria (Scenesse), and made the first recommendation worldwide of a stem-cell based therapy - Holoclar, for limbal stem cell deficiency - bringing new options in areas of high unmet need closer to patients.

The European Medicines Agency also highlighted that, overall, the number of medicines containing new active substances is on the rise, with 50% recommended for approval in 2014 containing a novel substance never been used in medicines before, in a strong show of R&D success.

Things are also looking good going forward, with a general increase in pre-authorisation activities in human-use medicines forecast for this year. In particular, a sustained increase in requests for scientific advice on clinical development, including parallel advice with health technology assessment (HTA) bodies, is expected, the Agency noted earlier this month.

*Three of the applications approved by the CHMP were withdrawn before a decision by the EMA was made.