Activities after orphan designation

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Orphan designation makes the sponsor eligible for a number of orphan incentives. Sponsors need to comply with various activities that take place after a designation has been granted.

Sponsors must submit an annual report on development to the Agency summarising the status of development of the medicine.

Sponsors of medicines with orphan designation should also remember to apply for a paediatric investigation plan (PIP), deferral or waiver at the appropriate time, as specified in the Paediatric regulation. For more information, see Rewards and incentives for paediatric medicines and PIP/waiver questions and answers.

Sponsors also need to submit an application for maintenance of the orphan designation at the time of marketing authorisation, in order to be eligible for the ten-year market exclusivity incentive. For more information, see Applying for marketing authorisation: orphan medicines.

A valid and completed paediatric investigation plan (PIP) could make the sponsor eligible for the two-year marketing exclusivity extension to the ten-year marketing exclusivity which is granted at the time of review of the orphan medicinal designation. For further information, see market exclusivity and refer to Questions and answers on the procedure of PIP compliance verification at EMA, and on paediatric rewards.

Transfers of orphan designation from one sponsor to another are possible. Transfers are free of charge.

Sponsors can also request removal of an orphan designation

Guidance for sponsors:

More information:

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