Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2015-08-10 | adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene | Laboratorios del Dr. Esteve (Spain) | mucopolysaccharidosis type II (Hunter's syndrome) |
Granting of the orphan status in the EU |
2015-08-10 | allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 | Fate Therapeutics (USA - CA) | acute lymphoblastic leukaemia |
Granting of the orphan status in the EU |
2014-04-10 | small molecule inhibitors of Rho-associated kinase (ROCK) including thiazovivin | Fate Therapeutics (USA - CA) | Granting of a patent | |
2015-10-11 | vadastuximab talirine CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA crosslinking pyrrolobenzodiazepine dimer drug | Seattle Genetics (USA - WA) | acute myeloid leukaemia |
Granting of the orphan status in the US |
2015-08-10 | fibrinogen-coated albumin spheres | Fibreu Limited (UK) | acute radiation syndrome |
Granting of the orphan status in the EU |
2015-08-10 | fixed-dose combination of fosfomycin disodium and tobramycin | CURx Pharma Limited (UK) | cystic fibrosis |
Granting of the orphan status in the EU |
2016-09-26 | ibrutinib | Janssen-Cilag International, a J&J company (USA - NJ) | marginal zone lymphoma |
Submission of an sNDA |
2015-08-10 | recombinant human acid ceramidase | Plexcera Therapeutics EU (Ireland) | cystic fibrosis |
Granting of the orphan status in the EU |
2015-08-10 | verucerfont - 3-(4-Methoxy-2-methylphenyl)-2,5-dimethyl-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5-yl)propyl]pyrazolo[1,5-a]pyrimidin-7-amine | Neurocrine Therapeutics (USA - CA) | congenital adrenal hyperplasia |
Granting of the orphan status in the EU |
2014-01-10 | Helix™ Transendocardial Delivery Catheter | Biocardia (USA - CA) | infusion of biologics to the heart |
Granting of a Market Authorisation in the EU |
2014-03-17 | Biocardia (USA - CA) | Granting of a Market Authorisation in the EU | ||
2015-06-16 | DNA plasmid encoding Stromal–cell Derived Factor 1 (SDF-1) | Juventas Therapeutics (USA - OH) | advanced ischemic chronic heart failure |
Granting of a Fast Track status |
2018-08-10 | migalastat (1-deoxygalactonojirimycin hydrochloride) | Amicus Therapeutics (USA - NJ) | Fabry disease | Granting of a Market Authorisation in the US |
2016-07-18 | fully human IgG2 monoclonal antibody that binds insulin receptors | Xoma (USA - CA) | congenital hyperinsulinism |
Granting of the orphan status in the EU |
2015-06-01 | docosahexaenoic acid | Sancilio (USA - FL) | short bowel syndrome |
Granting of the orphan status in the US |
2016-01-11 | synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol conjugated,acyclic nucleobase analogue | Arrowhead Research (USA - CA) Pharma Gateway (Sweden) | alpha-1 antitrypsin deficiency (AATD) |
Granting of the orphan status in the EU |
2015-06-01 | ibudilast | MediciNova (USA - CA) | Krabbe disease |
Granting of the orphan status in the US |
2015-07-31 | tipelukast | MediciNova (USA - CA) | NASH (non-alcoholic steatohepatitis) with fibrosis |
Granting of a patent |
2015-06-08 | adenoviral vector expressing the E.coli purine nucleoside phosphorylase gene (gedeptin) and fludarabine | PNP Therapeutics (USA - AL) | intratumoral treatment of anatomically accessible oral and pharyngeal cancers (lip, tongue, gum, floor of mouth, salivary gland, and other oral cavity) |
Granting of the orphan status in the US |
2015-05-27 | adult hemogenic endothelial cells | HemoGenyx (USA - NY) | aplastic anemia |
Granting of the orphan status in the US |
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