Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2017-06-15 | Bacillus subtilis oxalate decarboxylase | Allena Pharmaceuticals (USA - MA) | primary hyperoxaluria | Positive opinion for the granting of the orphan status in the EU |
2017-06-15 | retinol | Orphanix (Austria) | prevention of retinopathy of prematurity | Positive opinion for the granting of the orphan status in the EU |
2017-06-15 | polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1-3)-beta-D-galactopyranoside | SFL Regulatory Affairs Consulting (UK) | anti-MAG neuropathy | Positive opinion for the granting of a Market Authorisation in the EU |
2017-05-12 | decitabine and tetrahydrouridine | Ulrich Muehlner | sickle cell disease | Positive opinion for the granting of the orphan status in the EU |
2017-05-12 | ibutamoren mesilate | Richardson Associates Regulatory Affairs (UK) | growth hormone deficiency | Positive opinion for the granting of the orphan status in the EU |
2017-06-15 | sildenafil | Avivia Beheer (The Netnerlands) | congenital diaphragmatic hernia | Positive opinion for the granting of the orphan status in the EU |
2017-05-12 | sirolimus | Vale Pharmaceuticals (Ireland) | tuberous sclerosis | Positive opinion for the granting of the orphan status in the EU |
2017-05-12 | synthetic glucagon analogue modified to contain 7 amino acid substitutions | Zealand Pharma (Denmark) | congenital hyperinsulinism | Positive opinion for the granting of the orphan status in the EU |
2017-06-20 | tripotassium citrate monohydrate and potassium hydrogen carbonate | Advicenne Pharma (France) | distal renal tubular acidosis | Granting of the orphan status in the EU |
2017-05-12 | Asp-Arg-Val-Tyr-Ile-His-Pro | Envigo Pharma Consulting (UK) | epidermolysis bullosa | Positive opinion for the granting of the orphan status in the EU |
2017-05-12 | avacopan | ChemoCentryx (USA - CA) | C3 glomerulopathy | Positive opinion for the granting of the orphan status in the EU |
2017-05-12 | pentamer formyl thiophene acetic acid | NeuroScios (Austria) | Creutzfeldt-Jakob disease | Positive opinion for the granting of the orphan status in the EU |
2017-04-20 | autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains | Bluebird bio (USA - MA) | multiple myeloma | Granting of the orphan status in the EU |
2017-06-30 | polatuzumab vedotin | Roche (Switzerland) | relapsed or refractory diffuse large B cell lymphoma (DLBCL) | Granting of a Fast Track status |
2017-06-29 | Illumina (USA - CA) | colorectal cancer | Granting of a Market Authorisation in the US | |
2017-06-12 | adenovirus-associated viral vector serotype 5 containing the human RLBP1 gene | Horama (France) | retinitis punctata albescens due to RLBP1 gene mutation | Granting of the orphan status in the US |
2016-11-08 | mRNA-encoded antibodies, RNAntibody® technology. | Curevac (Germany) | Granting of a patent | |
2017-07-20 | ciclosporin | Santen (Japan) | severe vernal keratoconjunctivitis in children from 4 years of age and adolescents | Positive opinion for the granting of a Market Authorisation in the EU |
2017-07-20 | iloperidone | Vanda Pharmaceuticals (USA - WA) | schizophrenia | Negative opinion for the granting of a Market Authorisation in the EU |
2017-05-23 | plazomicin | Achaogen (USA - CA) | bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae (CRE) | Granting of the Breakthrough Therapy status |
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