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

Beraprost sodium

Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension 2001-09-18 Withdrawn  

Betaine anhydrous

Treatment of homocystinuria 2001-07-09 Withdrawn Cystadane


Treatment of idiopathic pulmonary fibrosis 2008-09-05 Withdrawn  


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


Treatment of systemic sclerosis (scleroderma) 2003-03-17 Withdrawn Tracleer


Treatment of chronic myeloid leukaemia 2010-08-04 Withdrawn Bosulif

Boswellia serrata resin dry extract

Treatment of peritumoral oedema derived from brain tumours 2002-10-21 Withdrawn  

Brivanib alaninate

Treatment of hepatocellular carcinoma 2011-10-27 Withdrawn  


Treatment of oesophageal cancer 2002-04-30 Withdrawn  


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

Carbamic acid (common)/[[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methyl-ethyl]phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester (amelubant)

Treatment of cystic fibrosis 2002-06-26 Withdrawn  

Carmustine (solution for intratumoral injection)

Treatment of glioma 2002-03-05 Withdrawn  

Chimeric anti-interleukin-6 monoclonal antibody

Treatment of multiple myeloma 2009-06-12 Withdrawn  

Chimeric anti-interleukin-6 monoclonal antibody

Treatment of renal cell carcinoma 2003-06-30 Withdrawn  

Chimeric monoclonal antibody against kappa myeloma antigen

Treatment of multiple myeloma 2012-05-22 Withdrawn  

Cholest-4-en-3-one, oxime

Treatment of amyotrophic lateral sclerosis 2006-08-28 Withdrawn  

Ciclosporin (implant)

Prevention of rejection for corneal transplant 2006-11-02 Withdrawn  

Ciclosporin (inhalation use)

Prevention of graft rejection after lung transplantation 2005-03-09 Withdrawn  


Treatment of atopic keratoconjunctivitis 2004-04-14 Withdrawn  


Treatment of glioma 2004-01-14 Withdrawn  


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

Colistimethate sodium

Treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis 2002-02-19 Withdrawn  

Complement factor H

Atypical haemolytic uraemic syndrome 2007-01-26 Withdrawn  

Cyclo {{(E,Z)-(2S,3R,4R)-3-hydroxy-4-methyl-2-(methylamino)nona-6,8-dienoyl}-L-2-aminobutyryl-N-methyl-glycyl-N-methyl-L-leucyl-L-valyl-N-methyl-L-leucyl-L-alanyl-D-alanyl-N-methyl-L-leucyl-N-methyl-L-leucyl-N-methyl-L-valyl}

Treatment of chronic non-infectious uveitis 2007-09-14 Withdrawn  


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