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-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione

Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes 2017-12-12 Positive  

2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib)

Treatment of acute myeloid leukaemia 2016-04-28 Positive  

2-{4-[(5,6-Diphenylpyrazin-2-yl)(isopropyl)amino]butoxy}-N-(methylsulfonyl)acetamide

Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension 2005-08-26 Withdrawn Uptravi

20% Intravenous fat emulsion consisting of 20% soybean oil, 1.2% egg yolk phospholipids, 2.25% glycerin, and water for injection

Treatment of poisoning by local anaesthetics 2017-04-03 Negative  

20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'-carboxylate

Treatment of ovarian cancer 2011-09-27 Positive  

26 base single stranded phosphodiester DNA oligonucleotide

Treatment of acute myeloid leukaemia 2009-10-08 Withdrawn  

26 base single stranded phosphodiester DNA oligonucleotide

Treatment of pancreatic cancer 2006-02-16 Withdrawn  

26 base single stranded phosphodiester DNA oligonucleotide

Treatment of renal cell carcinoma 2006-02-16 Withdrawn  

26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound

Treatment of Dravet syndrome 2017-02-27 Positive  

2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3'

Treatment of Huntington's disease 2015-02-12 Positive  

2S, 4R ketoconazole

Treatment of Cushing's syndrome 2012-07-04 Positive  

3,4 Diaminopyridine phosphate

Treatment of Lambert-Eaton myasthenic syndrome 2002-12-18 Positive Firdapse

3,5-diiodothyropropionic acid

Treatment of Allan-Herndon-Dudley syndrome 2013-10-07 Positive  

3-(3-Methanesulfonyl-phenyl)-1-propyl-piperidine hydrochloride

Treatment of narcolepsy 2016-07-13 Negative  

3-(4'Aminoisoindoline-1'-one)-1-piperidine-2,6-dione (lenalidomide)

Treatment of multiple myeloma 2003-12-12 Withdrawn Revlimid

3-(4'Aminoisoindoline-1'-one)-1-piperidine-2,6-dione

Treatment of myelodysplastic syndromes 2004-03-07 Positive Revlimid

3-(5-Amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride-

Treatment of diffuse large B-cell lymphoma 2016-06-27 Positive  

3-(6-(1-(2,2-Difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid (lumacaftor)

Treatment of cystic fibrosis 2010-08-04 Withdrawn Lumacaftor

3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone

Treatment of Rett syndrome 2014-02-19 Positive  

3-Pentylbenzeneacetic acid sodium salt

Treatment of idiopathic pulmonary fibrosis 2015-10-09 Positive  

3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid (avoralstat)

Treatment of hereditary angioedema 2015-02-12 Withdrawn  

3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt

Treatment of idiopathic pulmonary fibrosis 2016-07-14 Positive  

3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren)

Treatment of Duchenne muscular dystrophy 2005-05-27 Positive Translarna

3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid

Treatment of cystic fibrosis 2005-05-27 Positive  

3-methoxy-pregnenolone

Treatment of spinal cord injury 2007-12-04 Positive