EMA/CHMP recommends 12 new orphan medicines over the past year

Over the past 12 months, a total of 12 medicines for the treatment of rare diseases were recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).

They include medicines for the treatment of rare cancers (three), multidrug resistant tuberculosis (three) and pulmonary arterial hypertension (two), the EMA announced ahead of Rare Disease Day, which takes place on Friday (February 28), noting that about 30 million people living in the European Union suffer from a rare disease, which is a condition that does not affect more than five in 10,000 people.

The Agency plays an important role in the development and authorization of medicines for rare diseases, known as orphan medicines. Its Committee for Orphan Medicinal Products (COMP) issues recommendations to grant orphan designation to medicines, and marketing-authorization applications for designated orphan medicines are centrally assessed at the European level, rather than in each member state separately.