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

4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid

Treatment of cystic fibrosis 2014-11-19 Positive  

4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol (tanzisertib)

Treatment of idiopathic pulmonary fibrosis 2011-12-09 Withdrawn  

4-[[[4-(4-Chlorophenoxy)phenyl]sulfonyl]-methyl]tetrahydro-
N-hydroxy-2H-pyran-4-carboxamide

Prevention of graft rejection after liver transplantation 2008-09-10 Negative  

4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl)cyclopent-2-en-1-yl]pyrimidin-2-one

Treatment of pancreatic cancer 2017-10-12 Positive  

4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid 

Treatment of acute myeloid leukaemia 2014-08-22 Positive  

5’-CTG CCA CGT TCT CCT GC-(2’ methoxy)A-(2’ methoxy)C-(2’ methoxy)C-3’

Treatment of myasthenia gravis 2004-06-21 Positive  

5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine

Treatment of acute myeloid leukaemia 2007-09-14 Positive  

5'-O-(trans-9''-octadecenoyl)-1-beta-D-2'-deoxy-2',2'-difluorocytidine-

Treatment of pancreatic cancer 2009-10-28 Withdrawn  

5(S)-(2'-hydroxy ethoxy)-20(S)-Camptothecin

Treatment of osteosarcoma 2007-06-08 Positive  

5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin (redaporfin)

Treatment of biliary tract cancer 2015-03-19 Positive  

5,10-Methylene-tetrahydrofolic acid

Treatment of pancreatic cancer in combination with 5-fluorouracil 2004-09-02 Withdrawn  

5,5’-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol

Treatment of glioma 2014-12-16 Positive  

5,6,7,8-Tetrahydrobiopterin

Treatment of hyperphenylalaninemia 2003-10-02 Positive  

5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride

Treatment of Huntington’s disease 2015-05-21 Positive  

5-(2,6-Difluoro-phenoxy)-3(R,S)-{2(S)-[2(S)-(3-methoxycarbonyl-2(S)-{3-methyl-2(S)-[(quinoline-2-carbonyl)-amino]-butyrylamino}-propionylamino)-3-methyl-butyrylamino]-propionylamino}-4-oxo-pentanoic acid methyl ester

Treatment of neonatal brain injury 2006-10-23 Positive  

5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)pyridin-2-amine (bimiralisib)

Treatment of diffuse large B-cell lymphoma 2017-02-27 Positive  

5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid)

Treatment of Duchenne muscular dystrophy 2008-12-04 Positive  

5-10-Methylene-tetrahydrofolate

Treatment pancreatic cancer in combination with 5-fluorouracil 2003-06-11 Withdrawn  

5-Aminolevulinic acid hydrochloride

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

5-Methyl-pyridine-2-sulfonic acid{6-(2-hydroxy-ethoxy)-5-(2-methoxy-phenoxy)-2-[2-(1H-tetrazol-5-yl)-pyridin-4-yl]-pyrimidin-4-yl}-amide sodium salt

Treatment of aneurysmal subarachnoid haemorrhage 2003-12-11 Positive  

5-[1-(2,6-Dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride

Treatment of 5q spinal muscular atrophy 2011-08-30 Positive  

5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride

treatment of adrenoleukodystrophy 2016-11-18 Positive  

5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine

Treatment of malignant mesothelioma 2014-12-16 Withdrawn  

5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]

Treatment of biliary tract cancer 2017-10-16 Positive  

5-aminolevulinic acid

Treatment of glioma 2017-01-12 Positive -