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

Recombinant humanised monoclonal antibody against human complement component C5a

Treatment of graft-versus-host disease 2016-08-29 Withdrawn  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Treatment of Huntington's disease 2016-07-14 Withdrawn  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Prevention of graft-versus-host disease 2015-12-14 Withdrawn  


Treatment of nasopharyngeal carcinoma 2015-11-11 Withdrawn  

Sialic acid (also known as aceneuramic acid)

Treatment of GNE myopathy 2015-10-16 Withdrawn  

Dronabinol and cannabidiol

Treatment of glioma 2015-10-09 Withdrawn  

2’-deoxyguanosylyl-(3’,5’-phosphoryl)-2’-deoxythymidylyl-(3’,5’-phosphoryl)- 2’-deoxyguanosylyl-(3’,5’-phosphoryl)-2’-deoxycytidylyl-(3’,5’-phosphoryl)-2’-deoxycytidylyl -(3’,5’-phosphoryl)-2’-deoxycytidylyl-(3’,5’-phosphoryl)-2’-deoxyguanosylyl...

Treatment of diffuse large B-cell lymphoma 2015-08-10 Withdrawn  

Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2

Treatment of acute lymphoblastic leukaemia 2015-08-10 Withdrawn  


Treatment of congenital adrenal hyperplasia 2015-08-10 Withdrawn  

Synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene

Treatment of primary hyperoxaluria type 1 2015-07-28 Withdrawn  

Adeno-associated viral vector containing the human factor IX gene

Treatment of haemophilia B 2015-06-19 Withdrawn -


Treatment of marginal zone lymphoma 2015-06-19 Withdrawn -

Phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl) monohydrochloride

Treatment of Huntington’s disease 2015-04-24 Withdrawn  

Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene

Treatment of Gaucher disease 2015-03-19 Withdrawn  

Trientine tetrahydrochloride

Treatment of Wilson's disease 2015-03-19 Withdrawn  

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  


Treatment of sickle cell disease 2015-02-12 Withdrawn  


Treatment of narcolepsy 2015-02-12 Withdrawn  

Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells

Treatment of acute myeloid leukaemia 2015-01-15 Withdrawn  


Treatment of malignant mesothelioma 2014-12-16 Withdrawn  


Treatment of mucopolysaccharidosis type I 2014-11-19 Withdrawn  


Treatment of dermatomyositis 2014-11-19 Withdrawn  


Treatment of polymyositis 2014-11-19 Withdrawn  

(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  


Treatment of Schnitzler syndrome 2014-08-22 Withdrawn