Rare disease (orphan) designations

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This page allows you to find information on rare disease (orphan) designations based on applications that have been assessed by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP).

An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

For more information, see Orphan designation.

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EPAR Search results
Active substance Disease / condition Date of decision Decision Medicine name


Treatment of Smith-Magenis syndrome 2016-10-14 Positive  


Treatment of necrotising enterocolitis 2016-08-01 Positive  


Treatment of neonatal encephalopathy 2018-03-21 Positive  


Treatment of neonatal sepsis 2016-06-27 Positive  


Treatment of non-24-hour sleep-wake disorders in blind people with no light perception 2005-04-11 Withdrawn  


Treatment of partial deep dermal and full thickness burns 2018-03-13 Negative  


Treatment of perinatal asphyxia 2012-04-02 Positive