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

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Treatment of renal cell carcinoma 2005-03-09 Withdrawn Sutent

-[4-Aminobutanoic acid-glycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-L-aspartyl](cyclo 1-Dgamma17)

Treatment of pseudohypoaldosteronism 1B 2015-12-14 Positive  

1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane

Treatment to mobilize progenitor cells prior to stem cell transplantation 2004-10-20 Positive Mozobil

1,2-Bis(methylsulphonyl)-1-(2-chloroethyl)-2-[(methylamino)carbonyl]hydrazine

Treatment of acute myeloid leukaemia 2005-12-13 Positive  

1,2:5,6-Dianhydrogalactitol

Treatment of glioma 2013-01-24 Positive  

1,3-Propanedisulfonic acid, disodium salt

Treatment of systemic secondary amyloidosis 2001-07-31 Positive  

1,4-diamino-2,3-dicyano-1,4-bis[2-aminophenylthio] butadiene

Treatment of non-traumatic subarachnoid haemorrhage 2017-10-12 Positive  

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

Treatment of Gaucher disease 2000-10-18 Expired Zavesca

1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide (tezacaftor)

Treatment of cystic fibrosis 2014-07-04 Positive  

1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor

Treatment of cystic fibrosis 2017-02-27 Positive  

1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one

Treatment of multiple system atrophy 2014-12-16 Positive  

1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one

Treatment of Stargardt's disease 2018-05-25 Positive  

1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride

Treatment of cryptococcosis 2015-04-24 Positive  

1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride

Treatment of tularaemia 2015-04-24 Positive  

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-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor)

Treatment of idiopathic pulmonary fibrosis 2014-11-19 Positive  

1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor)

Treatment of systemic sclerosis 2014-11-19 Positive  

1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea

Treatment of acute myeloid leukaemia 2009-11-25 Positive  

1-Deoxygalactonojirimycin hydrochloride

Treatment of Fabry disease 2006-05-22 Positive  

1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene

Prevention of poliomyelitis in patients with immunodeficiencies deemed at risk 2012-07-17 Positive  

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib)

Treatment of chronic lymphocytic leukaemia 2012-04-26 Positive Imbruvica

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib)

Treatment of mantle-cell lymphoma 2013-03-12 Positive Imbruvica

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  

1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea

Ttreatment of gastrointestinal stromal tumours 2017-10-12 Positive