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

1,5-(Butylimino)-1,5-dideoxy, D-glucitol

Treatment of Gaucher disease 2000-10-18 Expired Zavesca

2-Chloro-9-[2-deoxy-2-fluoro-ß-D-arabinofuranosyl]adenine

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

5-Aminolevulinic acid hydrochloride

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

Alpha-galactosidase A

Treatment of Fabry disease 2000-08-08 Expired Replagal

Alpha-galactosidase A

Treatment of Fabry disease 2000-08-08 Expired Fabrazyme

Anagrelide hydrochloride

Treatment of essential thrombocythaemia 2000-12-29 Expired Xagrid

Arsenic trioxide

Treatment of acute promyelocytic leukaemia 2000-10-18 Expired Trisenox

Bosentan

Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension 2001-02-14 Expired Tracleer

Busulfan

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

Cladribine

Treatment of indolent non-Hodgkin’s lymphoma 2001-09-18 Expired Litak

Dasatinib

Treatment of acute lymphoblastic leukaemia 2005-12-23 Expired Sprycel

Dasatinib

Treatment of chronic myeloid leukaemia 2005-12-23 Expired Sprycel

Dexrazoxane

Treatment of anthracycline extravasations 2001-09-19 Expired Savene

Ecteinascidin 743 (trabectedin)

Treatment of soft tissue sarcoma 2001-05-30 Expired Yondelis

Hydroxyurea

Treatment of sickle cell syndrome 2003-07-09 Expired Siklos

Ibuprofen

Treatment of patent ductus arteriosus 2001-02-14 Expired Pedea

Iduronate-2-sulfatase

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

Iloprost

Treatment of primary and of the following forms of secondary pulmonary hypertension… 2000-12-29 Expired Ventavis

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