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

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  

3-pentylbenzeneacetic acid sodium salt

Treatment of Alström syndrome 2017-01-12 Positive -

3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid

Treatment of non-infectious uveitis 2015-06-19 Positive -

4’-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2’-(ethoxymethyl)-[1,1’-biphenyl]-2-sulfonamide

Treatment of focal segmental glomerulosclerosis 2015-11-11 Positive  

4,5-Dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid

Treatment of chronic iron overload requiring chelation therapy 2003-12-11 Withdrawn  

4,6,4’-trimethylangelicin

Treatment of cystic fibrosis 2013-06-19 Positive  

4,6,8-Trihydroxy-10-(3,7,11-trimethyldodeca-2,6,10-trienyl)-5,10-dihydrodibenzo[b,e][1,4] diazepin-11-one

Treatment of glioma 2009-05-15 Withdrawn  

4,7,10,13,16,19-docosahexaenoic acid

Treatment of retinitis pigmentosa 2006-11-03 Positive  

4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid

Treatment of chronic iron overload requiring chelation therapy 2002-03-13 Expired Exjade

4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid

Prevention of scarring post glaucoma filtration surgery 2014-06-04 Positive  

4-(4-{[2-(4-Chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide (venetoclax)

Treatment of chronic lymphocytic leukaemia 2012-12-06 Positive Venclyxto

4-Amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride

Treatment of moderate and severe traumatic brain injury 2006-08-28 Positive  

4-Amino-1-[5-O-[(2R,4S)-2-oxido-4-(4-pyridinyl)-1,3,2-dioxaphosphorinan-2-yl]-ß-D-arabinofuranosyl]-2(1H)-pyrimidinone

Treatment of hepatocellular carcinoma 2007-09-14 Positive  

4-Amino-5-oxo-4 (pyridinium-1-ylmethyl) proline

Treatment of renal cell carcinoma 2006-02-16 Positive  

4-Benzyl-2-naphtalen-1-yl-1,2,4-thiadiazolidine-3,5-dione

Treatment of progressive supranuclear palsy 2009-10-27 Withdrawn  

4-Ethoxy-2-(piperazin-1-yl)-7-(pyridin-4-yl)-5H-pyrimido[5,4-b]indol

Treatment of chronic lymphocytic leukaemia 2007-11-13 Positive  

4-Imino-1, 3-diazobicyclo-[3.1.0]-hexan-2-one

Treatment of pancreatic cancer 2005-07-27 Positive  

4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide

Treatment of retinitis pigmentosa 2016-05-30 Positive  

4-[123I] Iodo-L-phenylalanine

Diagnosis of glioma 2006-07-25 Positive  

4-[131I] Iodo-L-phenylalanine

Treatment of glioma 2006-04-11 Positive  

4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate

Treatment of hepatocellular carcinoma 2013-03-12 Positive  

4-[3,5-Bis(trimethylsilyl)benzamido] benzoic acid

Treatment of hepatocellular carcinoma 2007-10-22 Withdrawn  

4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1

Treatment of Huntington's disease 2005-06-20 Positive