Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-02-23 | riluzole | Biohaven Pharmaceutical (USA - CT) | spinocereballar ataxia |
Granting of the orphan status in the US |
2016-02-24 | sodium alginate oligosaccharide | Algipharma (Norway) | cystic fibrosis |
Granting of the orphan status in the US |
2016-02-04 | somatic cell therapy product containing hematopoietic progenitor cells (HPC), Facilitating Cells (FC), and alpha beta T cells. | Novartis (Switzerland) | prevention of organ rejection in living donor kidney transplant recipients |
Granting of the orphan status in the US |
2016-02-04 | synthesized peptide of the L-amino acids with the sequence H-Lys-Glu-Phe-Leu-His-Pro-Ser-Lys-Val-Asp-Leu-Pro-Arg-OH | Soricimed Biopharma (Canada) | ovarian cancer |
Granting of the orphan status in the US |
2016-06-29 | peptide consisting of L-amino acids with the sequence H-lys-glu-phe-leu-his-pro-ser-lys-val-asp-leu-pro-arg-OH | Soricimed Biopharma (Canada) | pancreatic cancer |
Granting of the orphan status in the US |
2016-03-10 | 1,2:5,6-dianhydrogalactitol | DelMar Pharmaceuticals (USA - CA) | medulloblastoma | Granting of the orphan status in the US |
2016-04-19 | 1,2:5,6-dianhydrogalactitol | DelMar Pharmaceuticals (USA - CA) | ovarian cancer | Granting of the orphan status in the US |
2016-04-20 | allogeneic ex-vivo generated CD56+CD3-natural killer cells from CD34+ umbilical cord blood progenitor cells | Glycostem-Therapeutics (The Netherlands) | acute myeloid leukemia |
Granting of the orphan status in the US |
2016-03-16 | adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA | Spark Therapeutics (USA - PA) | CLN2 disease (neuronal ceroid lipofuscinosis (NCL)) caused by TPP1 deficiency |
Granting of the orphan status in the US |
2017-12-07 | adenovirus associated viral vector serotype 5 containing the human RPE65 gene | MeiraGTx (USA - NY) | Leber congenital amaurosis | Granting of the orphan status in the US |
2018-06-20 | adenovirus associated viral vector serotype 8 containing the human CNGB3 gene | MeiraGTx (USA - NY) | achromatopsia due to mutations in the CNGB3 gene | Granting of the orphan status in the US |
2016-03-10 | anfibatide | Lee's Pharmaceutical (Hong Kong) (China) | thrombotic thrombocytopenic purpura |
Granting of the orphan status in the US |
2016-07-07 | (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide | Karyopharm Therapeutics (USA - MD) | soft tissue sarcoma |
Granting of the orphan status in the US |
2016-06-30 | small molecule Ewing Sarcoma breakpoint region 1/Friend leukemia virus integration 1 fusion protein inhibitor | Tokalas (USA - CA) now Oncternal Therapeutics (USA - CA) | Ewing sarcoma | Granting of the orphan status in the US |
2016-03-10 | bevacizumab | MicroSert (israel) | Coat's disease |
Granting of a patent |
2016-03-10 | competetive small molecule inhibitor of the enzyme O-GlcNAcase | Merck&Co (USA - NJ) | progressive supranuclear palsy |
Granting of the orphan status in the US |
2016-03-03 | encochleated amikacin | Aquarius Biotechnologies (USA - NJ) | non-tuberculous mycobacteria infections | Granting of the orphan status in the US |
2016-03-29 | gentamicin C2 congener | La Jolla Pharmaceuticals Company (USA - CA) | cystic fibrosis |
Granting of the orphan status in the US |
2016-03-17 | guanfacine and amphetamine | Genco Sciences (USA - IL) | Tourette's syndrome (0 through 16 years of age) | Granting of the orphan status in the US |
2016-03-31 | immune globulin infusion 10% (human) with recombinant human hyaluronidase) | Baxalta (USA - IL) | chronic inflammatory demyelinating polyneuropathy |
Granting of the orphan status in the US |
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