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

(-)-(2R)-3-(2-hydroxymethylindanyl-4-oxy)-phenyl-4,4,4-trifluorobutane-1-sulfonate

Treatment of moderate and severe closed traumatic brain injury 2008-09-05 Withdrawn  

(-)-trans-3-(5,6-dihydro-4H-pyrrolo[3,2,1-ij]quinolin-1yl)-4-(1H-indol-3-yl) pyrrolidine-2, 5-dione

Treatment of soft tissue sarcoma 2009-10-08 Withdrawn  

(1-Methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate

Treatment of soft-tissue sarcoma 2012-03-05 Withdrawn  

(1-Methyl-2-nitro-1H-imidazole-5-yl)methyl N,N’-bis(2-bromoethyl)diamidophosphate

Treatment of pancreatic cancer 2013-07-17 Withdrawn  

(1R, 2R, 4S)-4-{(2R)-2-[(3S,6R,7E,9R,10R,12R,14S,15E,17E,19E,21S,23S,26R, 27R,34aS)-9,27-dihydroxy-10,21-dimethoxy-6,8,12,14,20,26-hexamethyl-1,5,11,28,29-pentaoxo-1,4,5,6,9,10,11,12,13,14,21,22,23,24,25,26,27,28,29,31,32,33,34,34a-tetra-cosahydro-3H-23,2

Treatment of primary malignant bone tumours 2005-10-28 Withdrawn  

(1R, 2R, 4S)-4-{(2R)-2-[(3S,6R,7E,9R,10R,12R,14S,15E,17E,19E,21S,23S,26R, 27R,34aS)-9,27-dihydroxy-10,21-dimethoxy-6,8,12,14,20,26-hexamethyl-1,5,11,28,29-pentaoxo-1,4,5,6,9,10,11,12,13,14,21,22,23,24,25,26,27,28,29,31,32,33,34,34a-tetra-cosahydro-3H-23,2

Treatment of soft-tissue sarcoma 2005-08-26 Withdrawn  

(1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)-phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate

treatment of acute myeloid leukaemia 2010-03-23 Withdrawn  

(R)-3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-1-yl)-3-cyclopentylpropanenitrile phosphate

Treatment of chronic idiopathic myelofibrosis 2008-11-06 Withdrawn Jakavi

(R)-3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-1-yl)-3-cyclopentylpropanenitrile phosphate

Treatment of myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia 2009-04-03 Withdrawn Jakavi

(R, S)-3-(bromomethyl)-3-butanol-1-yl-disphosphate

Treatment of renal cell carcinoma 2004-07-29 Withdrawn  

(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile (tanziertib)

Treatment of pemphigus 2014-10-15 Withdrawn  

(Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate

Treatment of malignant gastrointestinal stromal tumours 2005-03-09 Withdrawn Sutent

(Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate

Treatment of renal cell carcinoma 2005-03-09 Withdrawn Sutent

1,5-(Butylimino)-1,5-dideoxy, D-glucitol

Treatment of Gaucher disease 2000-10-18 Expired Zavesca

1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea

Treatment of ovarian cancer 2012-06-06 Withdrawn  

1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea

Treatment of acute myeloid leukaemia 2011-10-27 Withdrawn  

1-[2-(Benzo[1,2,5]thiadiazol-5-ylamino)-6-(2,6-dichloro-phenyl)-pyrido[2,3-d]pyrimidin-7-yl]-3-tert-butyl-urea

Treatment of acute myeloid leukaemia 2010-09-20 Withdrawn  

17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride

Treatment of malignant gastrointestinal stromal tumours 2007-11-28 Withdrawn  

17-Allylamino-17-demethoxygeldanamycin

Treatment of chronic myeloid leukaemia 2005-01-26 Withdrawn  

17-Allylamino-17-demethoxygeldanamycin

Treatment of multiple myeloma 2004-12-21 Withdrawn  

2'-O-methyl-phosphorothioate oligonucleotide

Treatment of Duchenne muscular dystrophy 2006-02-15 Withdrawn  

2,3,4,5 Tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride

Treatment of Huntington's disease 2009-01-20 Withdrawn  

2-(4-(Diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine

Treatment of Duchenne muscular dystrophy 2006-07-25 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  

2-Chloro-9-[2-deoxy-2-fluoro-ß-D-arabinofuranosyl]adenine

Treatment of acute lymphoblastic leukaemia 2002-02-05 Expired Evoltra