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


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


Treatment of acute myeloid leukaemia 2003-05-08 Withdrawn  

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Treatment of Huntington's disease 2016-07-14 Withdrawn  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-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Prevention of graft-versus-host disease 2015-12-14 Withdrawn  


Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension 2005-08-26 Withdrawn Uptravi

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Treatment of acute myeloid leukaemia 2009-10-08 Withdrawn  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Treatment of pancreatic cancer 2006-02-16 Withdrawn  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Treatment of renal cell carcinoma 2006-02-16 Withdrawn  

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

Treatment of multiple myeloma 2003-12-12 Withdrawn Revlimid

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Treatment of cystic fibrosis 2010-08-04 Withdrawn Lumacaftor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-[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Treatment of hereditary angioedema 2015-02-12 Withdrawn  

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  

4,6,8-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-(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


Treatment of progressive supranuclear palsy 2009-10-27 Withdrawn  

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

Treatment of hepatocellular carcinoma 2007-10-22 Withdrawn  

4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol (tanzisertib)

Treatment of idiopathic pulmonary fibrosis 2011-12-09 Withdrawn  


Treatment of pancreatic cancer 2009-10-28 Withdrawn  

5,10-Methylene-tetrahydrofolic acid

Treatment of pancreatic cancer in combination with 5-fluorouracil 2004-09-02 Withdrawn  


Treatment pancreatic cancer in combination with 5-fluorouracil 2003-06-11 Withdrawn  

5-Aminolevulinic acid hydrochloride

Intra-operative photodynamic diagnosis of residual glioma 2002-11-13 Expired Gliolan


Treatment of malignant mesothelioma 2014-12-16 Withdrawn  


Treatment of sickle cell disease 2015-02-12 Withdrawn  

8-Cyclopentyl-1, 3-dipropylxanthine

Treatment of cystic fibrosis 2001-03-29 Withdrawn  

Streptococcus pyogenes Su strain cells treated with benzylpenicillin

Treatment of congenital lymphatic malformations 2010-01-29 Withdrawn