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


Adjunctive treatment to cytotoxic therapy in acute myeloid leukaemia 2011-12-09 Withdrawn  

Complement factor H

Atypical haemolytic uraemic syndrome 2007-01-26 Withdrawn  


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

Folic acid to be used with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine

Diagnosis of positive folate-receptor status in ovarian cancer 2012-09-10 Withdrawn Neocepri

N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine and folic acid

Diagnosis of positive folate-receptor status in ovarian cancer 2012-02-09 Withdrawn  

5-Aminolevulinic acid hydrochloride

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

Human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin

Late onset sepsis in premature infants of less or equal than 32 weeks of gestational age 2005-12-13 Withdrawn  


Patent ductus arteriosus in premature neonates of less than 34 weeks of gestational age 2001-03-05 Withdrawn  

Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins

Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection 2014-02-20 Withdrawn  

Ciclosporin (inhalation use)

Prevention of graft rejection after lung transplantation 2005-03-09 Withdrawn  

Murine IgM monoclonal antibody binding to alpha beta T-cell receptor

Prevention of graft rejection following solid organ transplantation 2013-03-12 Withdrawn  

2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy)phenyl]thio]phenyl]ethyl]-1,3-propanediol hydrochloride

Prevention of graft-versus-host disease 2015-12-14 Withdrawn  

Human monoclonal hepatitis B immunoglobulins (common)

Prevention of hepatitis B re-infection following liver transplantation 2004-02-22 Withdrawn  

Recombinant homodimer of the human annexin V

Prevention of ischaemia / reperfusion injury associated with solid-organ transplantation 2012-01-11 Withdrawn  

Purified inactivated Japanese encephalitis SA14-4-2 virus vaccine

Prevention of Japanese encephalitis 2006-01-24 Withdrawn  

Myristolated-peptidyl-recombinant SCR1-3 of human complement receptor type I

Prevention of post transplantation graft dysfunction 2002-07-30 Withdrawn  

Octavalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate

Prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis 2001-12-10 Withdrawn  

Ciclosporin (implant)

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

Human engineered monoclonal antibody specific for transforming growth factor β2

Prevention of scarring in glaucoma filtration surgical procedures 2001-05-30 Withdrawn  

Recombinant human serum amyloid P

Prevention of scarring post glaucoma filtration surgery 2009-10-09 Withdrawn  


Prevention of sepsis caused by Gram-positive pathogens in premature infants less than or equal to 34 2010-06-09 Withdrawn  

Anti-von Willebrand aptamer

Treament of thrombotic thrombocytopenic purpura 2008-06-03 Withdrawn  


Treatment idiopathic pulmonary fibrosis 2010-10-01 Withdrawn  

Imatinib mesilate

Treatment of dermatofibrosarcoma protuberans 2005-08-26 Expired Glivec

Pegylated recombinant factor VIIa

Treatment of haemophilia A 2008-06-04 Withdrawn