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 Medicines for rare diseases.

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EPAR Search results
Active substance Disease / condition Date of decision Decision Medicine name

Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors

Treatment of mantle cell lymphoma 20/12/2004 Positive  

Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors

Treatment of multiple myeloma 20/12/2004 Positive  

Recombinant homodimer of the human annexin V

Prevention of ischaemia / reperfusion injury associated with solid-organ transplantation 11/01/2012 Withdrawn  

Recombinant human ADAMTS-13

Treatment of thrombotic thrombocytopenic purpura 02/12/2008 Positive  

Recombinant human C1-inhibitor

Prevention of delayed graft function after solid organ transplantation 19/02/2007 Positive  

Recombinant human C1-inhibitor

Treatment of angioedema caused by C1 inhibitor deficiency 11/05/2001 Withdrawn  

Recombinant human CXCL8 mutant

Prevention of delayed graft function after solid organ transplantation 22/09/2008 Positive  

Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein

Treatment of hereditary angioedema 09/10/2015 Positive  

Recombinant human N-acetylgalactosamine-6-sulfatase

Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome) 24/07/2009 Positive Vimizim

Recombinant human acid alpha-glucosidase

Treatment of glycogen storage disease type II (Pompe's disease) 14/02/2001 Expired Myozyme

Recombinant human acid ceramidase

Treatment of Farber disease 19/02/2014 Positive  

Recombinant human acid ceramidase

Treatment of cystic fibrosis 10/08/2015 Positive  

Recombinant human acid sphingomyelinase

Treatment of Niemann-Pick disease, type B 19/09/2001 Positive  

Recombinant human alkaline phosphatase

Treatment of hypophosphatasia 15/01/2015 Positive  

Recombinant human alpha 1 chain homotrimer of type VII collagen

Treatment of epidermolysis bullosa 04/06/2014 Positive  

Recombinant human alpha-1 antitrypsin

Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency 30/04/2002 Withdrawn  

Recombinant human alpha-1 antitrypsin

Treatment of emphysema secondary to congenital alpha-1-antitrypsin deficiency 30/05/2001 Withdrawn  

Recombinant human alpha-1-microglobulin

Treatment of pre eclampsia 04/07/2014 Positive  

Recombinant human alpha-Mannosidase

Treatment of alpha-mannosidosis 26/01/2005 Positive  

Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan

Treatment of glycogen storage disease type II (Pompe's disease) 26/03/2014 Positive  

Recombinant human anti-interferon gamma monoclonal antibody

Treatment of haemophagocytic lymphohistiocytosis 09/06/2010 Positive  

Recombinant human apolipoprotein A-I in a complex with phospholipids

Treatment of ATP-binding cassette transporter A1 deficiency 22/08/2014 Positive  

Recombinant human apolipoprotein A-I in a complex with phospholipids

Treatment of apolipoprotein A-I deficiency 22/08/2014 Positive  

Recombinant human arylsulfatase A

Treatment of metachromatic leukodystrophy 09/07/2003 Withdrawn  

Recombinant human arylsulfatase A

treatment of metachromatic leukodystrophy 26/11/2010 Positive