Rare disease (orphan) designations

  • Email
  • Help

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.

Document status

Include:

Browse by letter

Search for active substance by letter and/or number:

Search by keyword

Search for keyword:

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

Abarelix

Treatment of low-flow priapism 2010-09-20 Withdrawn  

Abetimus sodium

Treatment of lupus nephritis 2001-11-20 Withdrawn  

Acetylcysteine

Treatment of idiopathic pulmonary fibrosis 2005-01-26 Withdrawn  

Adeno-associated viral vector containing the human alpha-sarcoglycan gene

Treatment of alpha-sarcoglycanopathy 2008-11-06 Withdrawn  

Adeno-associated viral vector containing the human calpain 3 gene

Treatment of calpainopathy 2006-04-06 Withdrawn  

Adeno-associated viral vector containing the human factor IX gene

Treatment of haemophilia B 2015-06-19 Withdrawn -

Adeno-associated viral vector containing the human gamma-sarcoglycan gene

Treatment of gamma-sarcoglycanopathy 2004-10-21 Withdrawn  

Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17-dimethylaminoethylamino-17-demethocygeldanamycin)

Treatment of retinitis pigmentosa 2012-11-08 Withdrawn  

Adeno-associated viral vector serotype 5 containing the human ABCA4 gene

Treatment of Stargardt's disease 2009-02-06 Withdrawn  

Adeno-associated viral vector serotype 8 containing the human AIPL1 gene

Treatment of Leber’s congenital amaurosis type 4 2011-12-09 Withdrawn  

Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene

Treatment of Gaucher disease 2015-03-19 Withdrawn  

Adenovirus-interferon gamma-coding DNA sequence

Treatment of cutaneous T-cell lymphoma 2003-07-09 Withdrawn  

Aldesleukin (inhalation use)

Treatment of renal cell carcinoma 2003-06-30 Withdrawn  

Alfimeprase

Treatment of acute peripheral arterial occlusion 2005-10-28 Withdrawn  

Alisertib

Treatment of ovarian cancer 2012-11-08 Withdrawn  

Alisertib

Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic / disseminated) 2012-12-06 Withdrawn  

Allogeneic bone marrow stem cells treated ex vivo with 16,16-dimethyl prostaglandin E2

Treatment of acute myeloid leukaemia 2011-05-13 Withdrawn  

Allogeneic ex-vivo-expanded umbilical-cord blood cells

Treatment of Hodgkin’s lymphoma 2009-07-24 Withdrawn  

Allogeneic ex-vivo-expanded umbilical-cord blood cells

Treatment of acute lymphoblastic leukaemia 2009-02-26 Withdrawn  

Allogeneic ex-vivo-expanded umbilical-cord blood cells

Treatment of acute myeloid leukaemia 2009-02-26 Withdrawn  

Allogeneic ex-vivo-expanded umbilical-cord blood cells

Treatment of chronic myeloid leukaemia 2009-10-08 Withdrawn  

Allogeneic ex-vivo-expanded umbilical-cord blood cells

Treatment of myelodysplastic syndromes 2009-10-08 Withdrawn  

Allogeneic human umbilical cord tissue-derived cells

Treatment of retinitis pigmentosa 2008-04-01 Withdrawn  

Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2

Treatment of acute lymphoblastic leukaemia 2015-08-10 Withdrawn  

Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells

Treatment of acute myeloid leukaemia 2015-01-15 Withdrawn