For help on how to get the results you want, see our search tips.
1713 results
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- for: Treatment of haemophilia B
Date of first decision: 14/12/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of carnitine palmitoyltransferase II deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of mitochondrial trifunctional protein deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of very long-chain acyl-CoA dehydrogenase deficiency
Date of first decision: 19/06/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec) for: treatment of glycogen storage disease type Ia
Date of first decision: 18/11/2016, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec) for: Treatment of ornithine transcarbamylase deficiency
Date of first decision: 21/03/2016, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of Prader-Willi syndrome
Date of first decision: 27/06/2016, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of first decision: 14/07/2016, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of first decision: 19/11/2018, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of Alström syndrome
Date of first decision: 09/01/2020, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome
Date of first decision: 21/08/2019, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease
Date of first decision: 11/11/2015, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt (niraparib) for: Treatment of ovarian cancer
Date of first decision: 04/08/2010, Positive, Last updated: 06/11/2020 -
List item
Orphan designation: Amikacin sulfate for: Treatment of nontuberculous mycobacterial lung disease
Date of first decision: 08/04/2014, Positive, Last updated: 03/11/2020 -
List item
Orphan designation: Amikacin sulfate for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of first decision: 25/07/2006, Positive, Last updated: 03/11/2020 -
List item
Orphan designation: 5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate for: Treatment of multiple system atrophy
Date of first decision: 16/12/2019, Positive, Last updated: 27/10/2020 -
List item
Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of first decision: 19/11/2014, Positive, Last updated: 27/10/2020 -
List item
Orphan designation: Arimoclomol for: Treatment of amyotrophic lateral sclerosis
Date of first decision: 26/10/2006, Positive, Last updated: 27/10/2020 -
List item
Orphan designation: arimoclomol citrate for: Treatment of inclusion body myositis
Date of first decision: 30/05/2016, Positive, Last updated: 27/10/2020 -
List item
Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 26/10/2020 -
List item
Orphan designation: Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/02/2020, Positive, Last updated: 22/10/2020 -
List item
Orphan designation: 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate for: Treatment of cystic fibrosis
Date of first decision: 26/10/2018, Positive, Last updated: 21/10/2020 -
List item
Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency
Date of first decision: 20/03/2007, Positive, Last updated: 20/10/2020 -
List item
Orphan designation: Magrolimab for: Treatment of myelodysplastic syndromes
Date of first decision: 26/06/2020, Positive, Last updated: 16/10/2020