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

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  

1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine, hydrochloride

Treatment of narcolepsy 2007-07-10 Positive  

11-(2-Pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Treatment of post-essential thrombocythaemia myelofibrosis 2010-08-25 Positive  

11-(2-Pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Treatment of post-polycythaemia vera myelofibrosis 2010-08-25 Positive  

11-(2-Pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Treatment of primary myelofibrosis 2010-08-25 Positive  

11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione

Treatment of cystic fibrosis 2014-02-19 Positive  

16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide

Treatment of neuroblastoma 2009-11-24 Positive  

16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide

Treatment of neuroblastoma 2012-07-04 Positive  

16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7 amino acid peptide

Treatment of soft tissue sarcoma 2016-07-14 Positive  

16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7-amino acid peptide

Treatment of medulloblastoma 2010-10-01 Positive  

17α,21-Dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione

Treatment of Duchenne muscular dystrophy 2014-08-22 Positive  

17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride

Treatment of malignant gastrointestinal stromal tumours 2007-11-28 Withdrawn  

17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5)

Treatment of retinitis pigmentosa 2012-11-28 Positive  

17-Allylamino-17-demethoxygeldanamycin

Treatment of chronic myeloid leukaemia 2005-01-26 Withdrawn  

17-Allylamino-17-demethoxygeldanamycin

Treatment of multiple myeloma 2004-12-21 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  

2',3',5'-Tri-O-acetyluridine

Treatment of 5-fluorouracil overdose 2009-05-15 Positive  

2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor

Treatment of acromegaly 2016-06-27 Positive  

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

Treatment of retinitis pigmentosa 2018-02-22 Positive  

2'-O-methyl-phosphorothioate oligonucleotide

Treatment of Duchenne muscular dystrophy 2006-02-15 Withdrawn  

2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid

Treatment of multiple myeloma 2011-09-27 Positive Ninlaro

2,2-Dimethylbutyric acid, sodium salt

Treatment of beta-thalassaemia intermedia and major 2009-02-26 Positive  

2,2-Dimethylbutyric acid, sodium salt

Treatment of sickle cell disease 2009-03-17 Positive  

2,3,4,5 Tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride

Treatment of Huntington's disease 2009-01-20 Withdrawn  

2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile

Treatment of idiopathic pulmonary fibrosis 2016-08-29 Positive