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

(E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt

Treatment of myelodysplastic syndromes 2012-04-26 Positive  

(Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-κN5, κN13, κN18, κN21, κN22]-)

Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy 2008-01-30 Positive  

(R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea-

Treatment of gastro-entero-pancreatic neuroendocrine tumours 2015-12-14 Positive  

(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate

Treatment of cystic fibrosis 2018-02-22 Positive  

(R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid)

Treatment of tuberculosis 2008-02-01 Positive Deltyba

(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Treatment of chronic idiopathic myelofibrosis 2008-11-06 Withdrawn Jakavi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)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride

Treatment of biliary tract cancer 2016-05-30 Positive  

(R)-troloxamide quinone

Treatment of amyotrophic lateral sclerosis 2017-10-12 Positive  

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

Treatment of renal cell carcinoma 2004-07-29 Withdrawn  

(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine  (avapritinib)

 

Treatment of gastrointestinal stromal tumours 2017-07-17 Positive  

(S)-10-[(dimethylamino)methyl]-4-ethyl-9-hydroxy-4-O-[alpha-(2", 4", 5", 7"-tetranitro-9"-fluorenylideneaminooxy)propionyl]-1H-pyrano[3', 4', 6', 7']indolizino[1,2-beta]-quinoline-3, 14-(4H, 12H)-dione, hydrochloride

Treatment of hepatocellular carcinoma 2010-10-01 Positive  

(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  

(S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine

Treatment of tuberculosis 2007-11-28 Positive  

(S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt (deferitazole)

Treatment of chronic iron overload requiring chelation therapy 2009-07-24 Positive  

(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one

Treatment of follicular lymphoma 2013-07-17 Positive  

(S)-6-Hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride

Treatment of Leigh syndrome 2014-10-15 Positive  

(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride

Treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes 2015-08-10 Positive  

(S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester

Treatment of pulmonary arterial hypertension 2017-04-20 Positive  

(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate (larotrectinib)

Treatment of soft tissue sarcoma 2016-01-11 Positive  

(S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate

Treatment of carcinoid syndrome 2016-05-30 Positive Xermelo

(S)-{8-Fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3, 4-dihydro-4-quinazolinyl} acetic acid (letermovir)

Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity deemed at... 2011-04-15 Positive  

(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide

Treatment of acute myeloid leukaemia 2014-08-22 Positive  

(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide

Treatment of diffuse large B-cell lymphoma 2014-08-22 Positive  

(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