Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2014-11-24 | Catalyst Pharmaceutical Partners (USA - FL) | Granting of a patent | ||
2015-05-21 | {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide | Right Track Regulatory Limited (UK) VentiRx Pharmaceuticals (USA - WA) | ovarian cancer |
Granting of the orphan status in the EU |
2015-05-21 | AASSGVSTPGSAGHDIITEQPRS (P42) | CNRS (France) | Huntington’s disease |
Granting of the orphan status in the EU |
2015-05-21 | adeno-associated viral vector serotype 9 containing the human HGSNAT gene | Cochamo Systems (UK) | mucopolysaccharidosis IIIC (Sanfilippo C syndrome) |
Granting of the orphan status in the EU |
2015-05-21 | genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain and a dimer of the D fragment from Staphylococcus aureus protein A | Toleranzia (Sweden) | myasthenia gravis |
Granting of the orphan status in the EU |
2015-05-21 | reduced oxydised N-acetyl heparin | Sigma-Tau Pharmaceuticals (USA - MD) Sigma-Tau Research Switzerland (Switzerland) | plasma cell myeloma/multiple myeloma |
Granting of the orphan status in the EU |
2015-05-21 | triamcinolone acetonide | S-cubed Limited (Denmark) | non-infectious uveitis |
Granting of the orphan status in the EU |
2015-05-21 | adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells | Regulatory Resources Group Ltd (UK) | thromboangiitis obliterans (Buerger\'s disease) |
Granting of the orphan status in the EU |
2015-05-21 | 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol- 6-yl)acetamide | Clinical Network Services (UK) | cystic fibrosis |
Granting of the orphan status in the EU |
2015-05-21 | allopurinol sodium | ACE Pharmaceuticals (The Netherlands) | perinatal asphyxia |
Granting of the orphan status in the EU |
2015-05-21 | humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 | ImmunoGen (USA - MA) | diffuse large B-cell lymphoma |
Granting of the orphan status in the EU |
2015-05-21 | trehalose | Dr Ulrich Granzer (Germany) | oculopharyngeal muscular dystrophy |
Granting of the orphan status in the EU |
2015-03-24 | amylopectin | Michael K. Davis, MD, MBA Nemours Children\'s Clinic (USA - FL) | glycogen storage disease types Ia and Ib |
Granting of the orphan status in the US |
2015-04-30 | antroquinonol | Golden Biotechnology Corporation (Taiwan) | acute myeloid leukemia |
Granting of the orphan status in the US |
2015-03-24 | avicin d | Avicin Therapeutics (USA - NC) | multiple myeloma |
Granting of the orphan status in the US |
2015-04-15 | [a-N-(2\'succinyl-paclitaxel)Thr]-Phe-Phe-Tyr-Gly-Gly-Ser-Arg-Gly- [epsilon-N-(2\'succinyl-paclitaxel)Lys]-Arg-Asn-Asn-Phe-[epsilon-N -(2\'succinyl-paclitaxel)Lys]-Thr-Glu-Glu-Tyr | Angiochem (Canada) | breast cancer patients with brain metastases |
Granting of the orphan status in the US |
2015-03-30 | bardoxolone | Reata Pharmaceuticals (USA - TX) | pulmonary arterial hypertension |
Granting of the orphan status in the US |
2015-03-31 | bryostatin 1 | Neurotrope BioScience (USA - NJ) | fragile X syndrome |
Granting of the orphan status in the US |
2015-04-21 | allogeneic cardiosphere-derived cells | Capricor Therapeutics (USA- CA) | cardiomyopathy associated with Duchenne muscular dystrophy |
Granting of the orphan status in the US |
2015-03-24 | allosteric activator of the red blood cell-specific form of pyruvate kinase | Agios Pharmaceuticals (USA -MA) | pyruvate kinase deficiency |
Granting of the orphan status in the US |
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