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

2-((3-((4-((3-aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5-dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3',4':6,7]naphtho[2,3-d][1,3]dioxol-5-yl)acetamide, tetrahydrochloride

Treatment of acute myeloid leukaemia 2015-07-28 Positive  

2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318) (olorofim)

Treatment of scedosporiosis 2016-08-29 Positive  

2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318)(olorofim)

Treatment of invasive aspergillosis 2016-10-14 Positive  

2-(2-Chlorobenzylidene)hydrazinecarboximidamide acetate-

Treatment of Charcot-Marie-Tooth disease 2015-12-14 Positive  

2-(2-Chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione

treatment of idiopathic pulmonary fibrosis 2010-11-26 Positive  

2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide

Treatment of small cell lung cancer 2014-08-22 Positive  

2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione

Treatment of systemic sclerosis 2015-10-09 Positive  

2-(2-phenylvinyl)-4-[4- methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt

Treatment of hepatocellular carcinoma 2015-08-10 Positive  

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-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide

Treatment of cystic fibrosis 2015-05-21 Positive  

2-Allyl-1-[6-(1-hydroxy-1-methylethyl)pyridin-2-yl]-6-{[4-(4-methylpiperazin-1-yl)phenyl]amino}-1,2-dihydro-3H-pyrazolo[3,4-d]pyrimidin-3-one

Treatment of ovarian cancer 2012-04-26 Positive  

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

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

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

Treatment of acute myeloid leukaemia 2003-05-08 Withdrawn  

2-Chloro-N6-(3-iodobenzyl)adenosine-5'-N-methyluronamide

Treatment of hepatocellular carcinoma 2015-10-09 Positive  

2-Ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate (remdesivir)

Treatment of Ebola virus disease 2016-02-17 Positive  

2-Hydroxyoleic acid

Treatment of glioma 2011-10-27 Positive  

2-Methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]-phenol

Treatment of anaplastic thyroid cancer 2004-04-14 Positive  

2-Methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one

treatment of acute myeloid leukaemia 2010-06-10 Positive  

2-[(2S)-2-methyl-1,4-dioxa-8-azaspiro[4.5]dec-8-yl]-8-nitro-6-trifluoromethyl-4H-1,3-benzothiazin-4-one

Treatment of tuberculosis 2018-06-27 Positive  

2-[4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid

Treatment of Huntington's disease 2016-07-14 Withdrawn  

2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid

Treatment of systemic sclerosis 2013-03-12 Positive  

2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine

Treatment of Charcot-Marie-Tooth disease 2017-10-16 Positive  

2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate

Treatment of acute myeloid leukaemia 2008-12-04 Positive  

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-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)

treatment of sickle cell disease 2016-11-18 Positive