Publication: Orphan designations

First reported 3 hours ago - Updated 3 hours ago - 1 reports

Orphan designation: Recombinant human apolipoprotein A-I in a complex with phospholipids, for the: Treatment of ATP-binding cassette transporter A1 deficiency

First reported 3 hours ago - Updated 3 hours ago - 1 reports

Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide, for the: Treatment of acute myeloid leukaemia

First reported 5 hours ago - Updated 5 hours ago - 1 reports

Orphan designation: Gevokizumab, for the: Treatment of Schnitzler syndrome

First reported 6 hours ago - Updated 6 hours ago - 1 reports

Orphan designation: (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate, for the: Treatment of Fabry disease

First reported Sep 29 2014 - Updated 17 hours ago - 1 reports

Orphan designation: Retinol, for the: Prevention of bronchopulmonary dysplasia

Entities: retinol
First reported Sep 29 2014 - Updated Sep 29 2014 - 1 reports

Orphan designation: Sodium ascorbate and menadione sodium bisulfite, for the: Treatment of autosomal dominant polycystic liver disease

Entities: Menadione, Sodium
First reported Sep 29 2014 - Updated Sep 29 2014 - 1 reports

Orphan designation: Lumacaftor/ivacaftor, for the: Treatment of cystic fibrosis

Entities: Cystic Fibrosis
First reported Sep 04 2014 - Updated Sep 04 2014 - 1 reports

Orphan designation: 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid, for the: Treatment of Duchenne muscular dystrophy

First reported Sep 01 2014 - Updated Sep 01 2014 - 1 reports

Orphan designation: Riociguat, for the: Treatment of systemic sclerosis

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Orphan designation: 5'-O-(trans-9 "-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine, for the: Treatment of acute myeloid leukaemia"

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Orphan designation: Recombinant human apolipopr... [Published Orphan designations - 3 hours ago]
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoro... [Published Orphan designations - 3 hours ago]
Orphan designation: Recombinant factor VIIa mod... [Published Orphan designations - 4 hours ago]
Orphan designation: Gevokizumab, for the: Treat... [Published Orphan designations - 5 hours ago]
Orphan designation: (3S)-1-azabicyclo[2.2.2]oct... [Published Orphan designations - 6 hours ago]
Orphan designation: Recombinant fusion protein ... [Published Orphan designations - Sep 29 2014]
Orphan designation: Sodium ascorbate and menadi... [Published Orphan designations - Sep 29 2014]
Orphan designation: Lumacaftor/ivacaftor, for t... [Published Orphan designations - Sep 29 2014]
Orphan designation: Retinol, for the: Preventio... [Published Orphan designations - Sep 29 2014]
Orphan designation: 3-[5-(2-Fluoro-phenyl)-[1,2... [Published Orphan designations - Sep 04 2014]
Orphan designation: Synthetic double-stranded s... [Published Orphan designations - Sep 01 2014]
Orphan designation: Riociguat, for the: Treatme... [Published Orphan designations - Sep 01 2014]
Orphan designation: Synthetic double-stranded s... [Published Orphan designations - Sep 01 2014]
Orphan designation: Recombinant fusion protein ... [Published Orphan designations - Sep 01 2014]
Orphan designation: Oxytocin, for the: Treatmen... [Published Orphan designations - Sep 01 2014]
Orphan designation: Cediranib, for the: Treatme... [Published Orphan designations - Sep 01 2014]
Orphan designation: Recombinant monoclonal anti... [Published Orphan designations - Sep 01 2014]
Orphan designation: Rilotumumab, for the: Treat... [Published Orphan designations - Sep 01 2014]
Orphan designation: Marizomib, for the: Treatme... [Published Orphan designations - Sep 01 2014]
Orphan designation: Cysteamine bitartrate, for ... [Published Orphan designations - Sep 01 2014]
Orphan designation: Humanised recombinant monoc... [Published Orphan designations - Sep 01 2014]
Orphan designation: Sodium acetate salt of the ... [Published Orphan designations - Sep 01 2014]
Orphan designation: Humanised anti-alpha ν... [Published Orphan designations - Sep 01 2014]
Orphan designation: Adeno-associated viral vect... [Published Orphan designations - Sep 01 2014]
Orphan designation: Eculizumab, for the: Treatm... [Published Orphan designations - Sep 01 2014]
Orphan designation: obinutuzumab, for the: Trea... [Published Orphan designations - Aug 22 2014]
Orphan designation: N-carbamyl-L-glutamic acid,... [Published Orphan designations - Jul 25 2014]
Orphan designation: Recombinant human alpha-1-m... [Published Orphan designations - Jul 16 2014]
Orphan designation: Norursodeoxycholic acid, fo... [Published Orphan designations - Jul 16 2014]
Orphan designation: Mixture of two adeno-associ... [Published Orphan designations - Jul 16 2014]
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Orphan designation: Recombinant human apolipopr... [Published Orphan designations - 3 hours ago]
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoro... [Published Orphan designations - 3 hours ago]
Orphan designation: Recombinant factor VIIa mod... [Published Orphan designations - 4 hours ago]
Orphan designation: Gevokizumab, for the: Treat... [Published Orphan designations - 5 hours ago]
Orphan designation: (3S)-1-azabicyclo[2.2.2]oct... [Published Orphan designations - 6 hours ago]
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