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

L-Asparaginase

Treatment of acute lymphoblastic leukaemia 2005-01-26 Withdrawn  

L-asparaginase encapsulated in erythrocytes

Treatment acute myeloid leukaemia 2013-02-08 Positive  

L-asparaginase encapsulated in erythrocytes

Treatment of acute lymphoblastic leukaemia 2006-10-27 Positive  

L-asparaginase encapsulated in erythrocytes

Treatment of pancreatic cancer 2009-05-15 Positive  

Pegylated L-asparaginase

Treatment of acute lymphoblastic leukaemia 2008-09-22 Positive  

Pegylated recombinant Erwinia chrysanthemi L-asparaginase

Treatment of acute lymphoblastic leukaemia 2011-08-05 Positive