EMA – European Medicines Agency has recently published a question-and-answer document useful to erase doubts related to the meaning of orphan designation and other aspects related to orphan medicines.

The EU legislation foresees giving orphan designation for substances that could be used for treating, preventing or diagnosing a rare and serious condition. Orphan designation can help the medicine’s developer advance the medicine to the stage where it can be authorised to be put on the market. A formal approval is needed before a medicine can legally be marketed.

The document also includes questions and answers related to significant benefits and market exclusivity of orphan medicines.

The EMA document on orphan designation is available here.