Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2014-11-13 | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Gamida Cell (Israel) | acute lymphoblastic leukemia |
Granting of the orphan status in the US |
2014-12-16 | allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media | Cell2B Advanced Therapeutics (Portugal) | prevention of graft-versus-host disease |
Granting of the orphan status in the EU |
2016-08-01 | pracinostat | Mei Pharma (USA - CA) | acute myeloid leulemia |
Granting of the Breakthrough Therapy status |
2014-11-13 | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Gamida Cell (Israel) | myelodysplastic syndrome |
Granting of the orphan status in the US |
2014-11-13 | stem/progenitor cells derived from ex vivo expanded allogeneic umbilical cord blood cells | Gamida Cell (Israel) | Hodgkin lymphoma |
Granting of the orphan status in the US |
2014-02-10 | Bruton\'s tyrosine kinase inhibitor | Celgene (USA - NJ) | chronic lymphocytic leukemia |
Granting of the orphan status in the US |
2014-11-17 | (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride | Khondrion (The Netherlands) | inherited mitochondrial respiratory chain diseases |
Granting of the orphan status in the US |
2015-01-07 | N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt | Atterocor (USA - MI) | Cushing's Syndrome |
Granting of the orphan status in the US |
2015-01-15 | 5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamide | Gilead Sciences (USA - CA) | pulmonary arterial hypertension |
Granting of the orphan status in the US |
2015-01-13 | pentapeptide with sequence Ac-VSRRR-NH2 | CereNOva (USA - NC) | intracerebral hemorrhage |
Granting of the orphan status in the US |
2015-01-15 | pentosan polysulfate sodium | Plexcera Therapeutics (USA - FL) | mucopolusaccharidosis type VI | Granting of the orphan status in the US |
2015-01-05 | simtuzumab | Gilead Sciences (USA - CA) | primary sclerosing cholangitis |
Granting of the orphan status in the US |
2018-06-14 | 4’-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2’- (ethoxymethyl)-[1,1’-biphenyl]-2-sulfonamide | Retrophin (USA - NY) | focal segmental glomerulosclerosis | Granting of a patent |
2015-01-15 | synthetic oligomer of 16 nucleotides | Isis Pharmaceuticals (USA - CA) | myotonic dystrophy Type I |
Granting of the orphan status in the US |
2015-01-05 | pyrvinium | StemSynergy Therapeutics (USA - FL) | familial adenomatous polyposis |
Granting of the orphan status in the US |
2015-01-06 | monoclonal antibody targeting eotaxin-2 | ChemomAb (Israel) | systemic sclerosis |
Granting of the orphan status in the US |
2015-02-12 | ulocuplumab | BMS (USA - NY) | acute myeloid leukemia |
Granting of the orphan status in the EU |
2015-01-13 | anti-Beta 1 integrin monoclonal antibody | Oncosynergy (USA - CA) | ovarian cancer |
Granting of the orphan status in the US |
2015-01-15 | autologous CD34+ bone marrow derived stem cells transduced with a self-inactivating gammaretroviral vector encoding the human IL2RG (yc) | Boston Children\'s Hospital Div. of Hematology/Oncology (USA - MA) | severe combined immune deficiency-X1 |
Granting of the orphan status in the US |
2015-01-13 | deutetrabenazine | Auspex Pharmaceuticals (USA - CA) now Teva Pharmaceutical Industries (Israel) | Tourette syndrome in the pediatric population | Granting of the orphan status in the US |
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