Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-03-23 | ivabradine | Amgen (USA - CA) | pediatric patients (0 through 16 years of age) with dilated cardiomyopathy |
Granting of the orphan status in the US |
2016-05-02 | Fancalen (lentiviral vector containing the Fanconi anemia-A (FANCA) gene | Centro de Investigacion Biomedica en Red (CIBER) (Spain) | Fanconi anemia type A patients |
Granting of the orphan status in the US |
2017-11-16 | autologous T-lymphocytes transduced with anti-BCMA02 CAR lentiviral vector encoding a chimeric antigen receptor (CAR) specific for the human B cell maturation antigen | Celgene (USA - NJ) Bluebird bio (USA - MA) | multiple myeloma | Granting of the Breakthrough Therapy status |
2016-03-10 | rosomidnar | ProNAi Therapeutics (USA - CA) | diffuse large B-cell lymphoma |
Granting of the orphan status in the US |
2016-03-03 | selective inhibitor of fungal CYP51 | Viamet Pharmaceuticals (USA - NC) | coccidioidomycosis |
Granting of the orphan status in the US |
2016-03-01 | tesevatinib | Kadmon Corporation (USA - NY) | patients with autosomal recessive polycystic kidney disease (ARPKD) |
Granting of the orphan status in the US |
2016-04-25 | (3S)-3-[({1-(2-Chlorobenzyl)-4-hydroxy-5-methyl-2-oxo-1,2-dihydropyridin-3yl}amino)carbonyl]amino-3-(4-methylphenyl)propanoic acid monosodium salt | Aviara Pharmaceuticals (USA - TX) | mobilization of hematopoietic stem cells |
Granting of the orphan status in the US |
2016-05-18 | anti-HER2 bispecific antibody | Zymeworks (Canada) | ovarian cancer |
Granting of a patent |
2016-07-06 | anti-HER2 bispecific antibody drug conjugate | Zymeworks (Canada) | Granting of the orphan status in the US | |
2016-08-18 | folate binding protein peptide: Glu-Ile-Trp-Thr-His-Ser-Tyr-Lys-Val | Galena BioPharma (USA - OR) | treatment (including prevention of recurrence) of ovarian cancer |
Granting of the orphan status in the US |
2016-08-18 | folate binding protein peptide: Glu-Ile-Trp-Thr-His-Ser-Tyr-Lys-Val | Galena BioPharma (USA - OR) | treatment (including prevention of recurrence) of ovarian cancer |
Granting of the orphan status in the US |
2016-05-23 | nanocomplex consisting of a small interfering RNA that targets the human EV11 oncogenic transcription factor and an elastin-like polypeptide drug carrier. | PeptiMed (USA - WI) | ovarian cancer |
Granting of the orphan status in the US |
2016-06-21 | sperm protein-17 oral microparticle vaccine | Kiromic (USA - TX) | Granting of the orphan status in the US | |
2016-04-05 | poly-N,N'-hexamethyleneguanidine-poly-N1,N4-hexamethyleneaminoguanidine | TGV-Inhalonix (USA - NY) | pulmonary infections in patients with cystic fibrosis including infections due to Pseudomonas aeruginosa,Burkholderia cepacia and other bacteria |
Granting of the orphan status in the US |
2016-04-21 | (E, Z)-4,5,9-trithiadodeca-1, 6, 11-triene 9-oxide (Ajoene) | Neem Biotech (UK) | Pseudomonas aeruginosa pulmonary infections in cystic fibrosis patients |
Granting of the orphan status in the US |
2016-04-06 | 1-(2,8-Dimethyl-1-thia-3,8-diazaspiro[4.5]dec-3-yl)-3-(1H-indol-3-yl)propan-1-one | Anavex Life Sciences (USA - NY) | frontotemporal dementia |
Granting of the orphan status in the US |
2016-04-05 | cerebrolysin | EVER Neuro Pharma (Austria) | frontotemporal dementia, including all subvariants |
Granting of the orphan status in the US |
2016-04-13 | 7-ethyl-10-hydroxycamptothecin (EHC) | Nippon Kayaku (Japan) | small cell lung cancer |
Granting of the orphan status in the US |
2016-04-19 | adenovirus encoding the recombinant CD40 ligand | Memgen (USA - TX) | stage IIb through IV melanoma |
Granting of the orphan status in the US |
2016-04-20 | aphidicolin | Biospherics.net (USA - KY) | Ebola virus infection |
Granting of the orphan status in the US |
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