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

Adeno-associated viral vector containing the human factor IX gene

Treatment of haemophilia B 2015-06-19 Withdrawn -


Treatment of marginal zone lymphoma 2015-06-19 Withdrawn -


Treatment of renal cell carcinoma 2007-06-05 Withdrawn Afinitor


Treatment of mucopolysaccharidosis type I 2001-02-14 Expired Aldurazyme


Treatment of acute lymphoblastic leukaemia 2005-06-16 Expired Atriance


Treatment of chronic myeloid leukaemia 2010-08-04 Withdrawn Bosulif


Conditioning treatment prior to haematopoietic-progenitor-cell transplantation 2000-12-29 Expired Busilvex

N-carbamyl-L-glutamic acid

Treatment of N-acetylglutamate synthetase (NAGS) deficiency 2000-10-18 Expired Carbaglu

Betaine anhydrous

Treatment of homocystinuria 2001-07-09 Withdrawn Cystadane


Treatment of severe myoclonic epilepsy in infancy 2001-12-05 Expired Diacomit


Treatment of mucopolysaccharidosis, type II (Hunter Syndrome) 2001-12-11 Expired Elaprase

Recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1 (efmoroctocog alfa)

Treatment of haemophilia A 2010-09-20 Withdrawn Elocta


Treatment of acute lymphoblastic leukaemia 2002-02-05 Expired Evoltra

4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid

Treatment of chronic iron overload requiring chelation therapy 2002-03-13 Expired Exjade

Alpha-galactosidase A

Treatment of Fabry disease 2000-08-08 Expired Fabrazyme

5-Aminolevulinic acid hydrochloride

Intra-operative photodynamic diagnosis of residual glioma 2002-11-13 Expired Gliolan

Imatinib mesilate

Treatment of dermatofibrosarcoma protuberans 2005-08-26 Expired Glivec

Imatinib mesilate

Treatment of acute lymphoblastic leukaemia 2005-08-26 Expired Glivec

Imatinib mesilate

Treatment of chronic eosinophilic leukaemia and the hypereosinophilic syndrome 2005-10-28 Expired Glivec

Imatinib mesilate

Treatment of chronic myeloid leukaemia 2001-02-14 Expired Glivec

Imatinib mesilate

Treatment of malignant gastrointestinal stromal tumours 2001-11-20 Expired Glivec

Imatinib mesilate

Treatment of myelodysplastic / myeloproliferative diseases 2005-12-23 Expired Glivec

Recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class

Treatment of cryopirin-associated periodic syndromes (FCUS, MWS, and NOMID) 2007-03-20 Withdrawn Ilaris


Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects 2006-05-22 Expired Increlex


Treatment of renal-cell carcinoma 2011-02-23 Withdrawn Inlyta