Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-10-18 | 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate | Coté Orphan Consulting UK (UK)/Eloxx Pharmaceuticals (Israel) | mucopolysaccharidosis type I |
Granting of the orphan status in the US |
2017-05-22 | adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene | Pfizer (USA - NY) | Duchenne muscular dystrophy | Granting of the orphan status in the US |
2016-08-29 | adenovirus associated viral vector serotype 5 containing the human RPGR gene | Athena Vision (UK) | retinitis pigmentosa |
Granting of the orphan status in the EU |
2016-08-29 | cisplatin | PlumeStars (Italy) | malignant mesothelioma |
Granting of the orphan status in the EU |
2016-08-29 | fimaporfin | PCI Biotech (Norway) | cholangiocarcinoma (bile duct cancer) |
Granting of the orphan status in the EU |
2016-08-29 | L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser | Araim Pharma Europe (UK) | graft loss in pancreatic islet transplantation |
Granting of the orphan status in the EU |
2016-08-29 | methotrexate | aimAKU (Associazione Italiana Malati di Alcaptonuria) (Italy) | alkaptonuria |
Granting of the orphan status in the EU |
2016-08-29 | nintedanib | Boehringer Ingelheim (Germany) | systemic sclerosis |
Granting of the orphan status in the EU |
2016-08-29 | recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues | NanoMedSyn (France) | Pompe's disease |
Granting of the orphan status in the EU |
2016-08-29 | recombinant human interleukin-12 | Coté Orphan Consulting UK (UK) | acute radiation syndrome |
Granting of the orphan status in the EU |
2016-08-29 | recombinant humanised monoclonal antibody against human complement component C5a | Alexion Pharmaceuticals (USA - CT) | graft-versus-host disease |
Granting of the orphan status in the EU |
2016-08-29 | synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acetylgalactosamine residues | Alnylam Therapeutics (USA - MA) | acute hepatic porphyria |
Granting of the orphan status in the EU |
2016-08-29 | synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid | Biogen (USA - MA) | amyotrophic lateral sclerosis |
Granting of the orphan status in the EU |
2016-08-29 | temozolomide | Double Bond Pharmaceutical (Sweden) | glioma |
Granting of the orphan status in the EU |
2016-06-16 | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Wave LIfe Sciences (USA - MA) | Huntington's disease |
Granting of the orphan status in the US |
2016-06-30 | autologous osteogenic cells cultured from adipose tissue derived stem cells embedded in extracellular matrix with demineralized bone matrix | NOvadip Biosciences (Belgium) | congenital pseudarthrosis of the tibia |
Granting of the orphan status in the US |
2016-06-20 | recombinant factor VIIa | Catalyst Biosciences (USA - CA) | severe hemophilia A and B with inhibitors |
Granting of a patent |
2016-06-20 | Catalyst Biosciences (USA - CA) | severe hemophilia B |
Granting of a patent | |
2016-06-20 | Catalyst Biosciences (USA - CA) | renal delayed graft function (DGF) and other ischemia reperfusion driven diseases age-related macular degeneration |
Granting of a patent | |
2016-08-04 | bacteriophage preparations followed by the antibiotic | Ampliphi Bioscience (USA - VA) | antibiotic-resistant infections |
Granting of a patent |
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