Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-07-18 | sirolimus | Best Regulatory Consulting (UK) | sporadic lymphangioleiomyomatosis |
Granting of the orphan status in the EU |
2016-07-18 | 16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7 amino acid peptide | Biogenera (Italy) | soft tissue sarcoma |
Positive opinion for the granting of the orphan status in the EU |
2016-07-18 | 2-[4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid | Pfizer (USA - NY) | Huntington's disease |
Granting of the orphan status in the EU |
2016-07-18 | autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity enhanced T-cell receptor targeting the New York esophageal antigen-1 (NY-ESO-1) | Adaptimmune Therapeutics (UK) | soft tissue sarcoma |
Granting of the orphan status in the EU |
2016-07-18 | autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo | Cell Medica (UK) | extranodal NK/T-cell lymphoma, nasal type |
Granting of the orphan status in the EU |
2016-07-18 | autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo | Cell Medica (UK) | post-transplant lymphoproliferative disorder. |
Granting of the orphan status in the EU |
2016-07-18 | brincidofovir | Chimerix (USA - NC) | adenovirus infection in immunocompromised patients |
Granting of the orphan status in the EU |
2016-07-18 | mifamurtide | Delta Proteomics (France) | echinococcosis |
Granting of the orphan status in the EU |
2016-07-18 | mifamurtide | Delta Proteomics (France) | hepatocellular carcinoma |
Granting of the orphan status in the EU |
2016-07-18 | setmelanotide | Rhythm Pharmaceuticals (USA - MA)/TMC Pharma Services (UK) | pro-opiomelanocortin deficiency (POMC) |
Granting of the orphan status in the EU |
2016-07-18 | sodium benzoate | Lucane Pharma (France) | carbamoyl-phosphate synthetase-1 deficiency |
Granting of the orphan status in the EU |
2016-07-18 | sodium benzoate | Lucane Pharma (France) | citrullinaemia type 1 |
Granting of the orphan status in the EU |
2016-07-18 | sodium benzoate | Lucane Pharma (France) | ornithine transcarbamylase deficiency |
Granting of the orphan status in the EU |
2016-07-18 | sodium hypochlorite | Hypo-Stream (UK) | partial deep dermal and full thickness burns |
Granting of the orphan status in the EU |
2016-07-18 | phosphorothioate oligonucleotide targeted to apolipoprotein C-III | Isis Pharmaceuticals (USA - CA), now Ionis Pharmaceuticals (USA - CA) | familial partial lipodystrophy | Granting of the orphan status in the EU |
2016-07-06 | seribantumab | Merrimack Pharmaceuticals (USA - MA) | heregulin-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC) whose disease has progressed following immunotherapy |
Granting of a patent |
2016-05-30 | novel macrocyclic compounds and their use as inhibitors of LRRK2 (Leucine-Rich Repeat Kinase 2) | Oncodesign (France) | Granting of a patent | |
2016-04-27 | 6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione | Myokardia (USA - CA) | symptomatic obstructive hypertrophic cardiomyopathy |
Granting of the orphan status in the US |
2016-06-23 | (S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydroprazolo[1,5-a]pyrimidine-3-carboxamide | BeiGene USA | mantle cell lymphoma |
Granting of the orphan status in the US |
2016-06-29 | (S)-7-(1-acryloylpiperidin-4-yl)-2-(4-phenoxyphenyl)-4,5,6,7-tetrahydropyrazolo[1,5-a]pyrimidine-3-carboxamide | BeiGene USA | Waldenstrom's macroglobulinemia | Granting of a Fast Track status |
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