Orphan Medicines in Europe

Navigating the Landscape of Orphan Medicines in the European Union

Finding an effective treatment for a patient suffering from one out of the more than 6000 distinct rare diseases in the EU can be very challenging. An orphan medicine is a medicinal product developed for the treatment of a rare disease. The EU’s orphan designation program encourages the development of medicines to help these patients.1

Over 1900 orphan medicines with designation

Advancing Treatment Options: Over 1900 Orphan Medicines Under Evaluation by the COMP

The Committee for Orphan Medicinal Products (COMP) is in charge for assessing applications for designation as an orphan medicinal product. If a medicine reaches the marketing authorization stage, it is re-evaluated by the COMP whether the criteria are still met and the orphan medicine status can be maintained.

Over 140 orphan medicines authorized in the EU

Milestones in Rare Disease Treatment: Over 140 Orphan Medicines Authorized in the EU

Medicinal products designated and authorized as orphan medicinal products receive market protection for 10 years.