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

Melatonin

Treatment of partial deep dermal and full thickness burns 2018-03-13 Negative  

(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate

Treatment of cystic fibrosis 2018-02-22 Positive  

1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride

Treatment of soft tissue sarcoma 2018-02-22 Positive  

2'-O-(2-Methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene

Treatment of retinitis pigmentosa 2018-02-22 Positive  

6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate

Treatment of acute myeloid leukaemia 2018-02-22 Positive  

Adenovirus-associated viral vector serotype 8 containing the human RPGR gene

Treatment of retinitis pigmentosa 2018-02-22 Positive  

Allogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120

Treatment in haematopoietic stem cell transplantation 2018-02-22 Positive  

Cannabidivarin

Treatment of fragile X syndrome 2018-02-22 Positive  

Flucytosine

Treatment of glioma 2018-02-22 Positive  

Human monoclonal IgG2 antibody against tissue factor pathway inhibitor

Treatment of haemophilia A 2018-02-22 Positive  

Levosimendan

Treatment of amyotrophic lateral sclerosis 2018-02-22 Positive  

Mertansine functionalised gold nanoconjugate

Treatment of hepatocellular carcinoma 2018-02-22 Positive  

N-(tert-butylcarbamoyl)-5-cyano-2-((4'-(difluoromethoxy)-[1,1'-biphenyl]-3-yl)oxy)benzenesulfonamide

Treatment of pulmonary arterial hypertension 2018-02-22 Positive  

Pyridoxal 5'-phosphate

Treatment of pyridoxamine 5'-phosphate oxidase deficiency 2018-02-22 Positive  

Recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2

Treatment of primary IgA nephropathy 2018-02-22 Positive  

Rusalatide acetate

Treatment of acute radiation syndrome 2018-02-22 Positive  

Seletalisib

Treatment of activated phosphoinositide 3-kinase delta syndrome 2018-02-22 Positive  

Vocimagene amiretrorepvec

Treatment of glioma 2018-02-22 Positive  

Vatiquinone

Treatment of RARS2 syndrome 2018-01-17 Positive  

Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene

Treatment of mucopolysaccharidosis type I 2018-01-17 Positive  

Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene

Treatment of mucopolysaccharidosis type II (Hunter's syndrome) 2018-01-17 Positive  

Adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene

Treatment of Huntington's disease 2018-01-17 Positive  

Allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1

Treatment in haematopoietic stem cell transplantation 2018-01-17 Positive  

Cannabidiol

Treatment of tuberous sclerosis 2018-01-17 Positive  

Ciclopirox

Treatment of congenital erythropoietic porphyria 2018-01-17 Positive