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

Human anthrax immunoglobulin

Treatment of inhalation anthrax disease 2009-11-17 Withdrawn  

Human anthrax monoclonal antibody

Treatment of inhalation anthrax disease 2011-04-15 Withdrawn  

Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh

Treatment of epidermolysis bullosa 2011-06-21 Withdrawn  

Human engineered monoclonal antibody specific for transforming growth factor β2

Prevention of scarring in glaucoma filtration surgical procedures 2001-05-30 Withdrawn  

Human engineered monoclonal antibody specific for transforming growth factor ß1

Treatment of systemic sclerosis 2002-02-04 Withdrawn  

Human immunoglobulin

Treatment of dermatomyositis 2003-10-20 Withdrawn  

Human immunoglobulin

Treatment of polymyositis 2003-10-24 Withdrawn  

Human monoclonal antibody against HLA-DR

Treatment of Hodgkin's lymphoma 2005-06-16 Withdrawn  

Human monoclonal antibody against HLA-DR

Treatment of Multiple Myeloma 2006-02-27 Withdrawn  

Human monoclonal antibody against HLA-DR

Treatment of chronic lymphocytic leukaemia 2006-01-24 Withdrawn  

Human monoclonal antibody against inhibitory killer cell lg-like receptors (1-7 F9)

Treatment of acute myeloid leukemia 2006-08-28 Withdrawn  

Human monoclonal hepatitis B immunoglobulins (common)

Prevention of hepatitis B re-infection following liver transplantation 2004-02-22 Withdrawn  

Human plasmin

Treatment of acute peripheral arterial occlusion 2011-02-23 Withdrawn  

Human transferrin conjugated to mutant diptheria toxin

Treatment of glioma 2002-03-19 Withdrawn  

Humanised anti-HM1.24 monoclonal antibody

Treatment of multiple myeloma 2001-05-10 Withdrawn  

Humanised monoclonal antibody against epidermal-growth-factor receptor

Treatment of glioma 2012-08-09 Withdrawn  

Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa (emicizumab)

Treatment of haemophilia A 2014-01-16 Withdrawn  

Humanized anti-KSA monoclonal antibody - human interleukin-2-fusion protein

Treatment of renal cell carcinoma 2002-03-22 Withdrawn  

Hydroxyurea

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

Ibuprofen

Patent ductus arteriosus in premature neonates of less than 34 weeks of gestational age 2001-03-05 Withdrawn  

Ibuprofen

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

Idelalisib

Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma 2013-08-05 Withdrawn  

Idelalisib

Treatment of extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma) 2013-08-05 Withdrawn  

Idelalisib

Treatment of follicular lymphoma 2013-07-17 Withdrawn  

Idelalisib

Treatment of lymphoplasmacytic lymphoma 2013-07-17 Withdrawn