Date |
Product |
Disease |
Phase |
Company |
Therapeutic area |
2017-12-10
|
avapritinib (BLU-285) |
advanced systemic mastocytosis |
1 |
Blueprint Medicines (USA - MA) |
Rare diseases - Hematological diseases |
2017-12-10
|
Elocta®/Eloctate® (long-lasting recombinant Factor VIII Fc fusion protein - rFVIIIFc - efmoroctocog alfa) |
hemophilia A |
3 |
Bioverativ (USA - CA) Swedish Orphan Biovitrum SOBI (Sweden) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-10
|
Yescarta™ (axicabtagene ciloleucel) (KTE-C19) (autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor) |
refractory diffuse large B cell lymphoma, refractory primary mediastinal B cell lymphoma, refractory transformed follicular lymphoma |
1-2 |
Kite Pharma (USA - CA) |
Cancer - Oncology - Rare diseases |
2017-12-10
|
Adcetris® (brentuximab vedotin) |
Hodgkin lymphoma |
3 |
Seattle Genetics (USA - WA) |
Cancer - Oncology |
2017-12-10
|
Elocta™/Eloctate™ (recombinant factor VIII Fc fusion protein - rFVIIIFc) |
hemophilia A |
3 |
BiogenIdec (USA) Swedish Orphan Biovitrum SOBI (Sweden) |
Hematologic diseases - Genetic diseases |
2017-12-09
|
Sprycel® (dasatinib) |
newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) |
2 |
BMS (USA - NY) |
Cancer - Oncology |
2017-12-09
|
chimeric antigen receptor (CAR)-targeted CD8alphabeta+ T cells |
|
preclinical |
Fate Therapeutics (USA - CA) |
Cancer - Oncology |
2017-12-09
|
Adcetris® (brentuximab vedotin) |
CD30-expressing cutaneous T-cell lymphoma (CTCL) |
3 |
Millenium Pharmaceuticals (USA - MA) Seattle Genetics (USA - WA), wholly owned subsidiaries of Takeda Pharmaceutical (Japan) |
Cancer - Oncology |
2017-12-09
|
IMGN779 |
acute myeloid leukemia, refractory acute myeloid leukemia |
1 |
immunogen (USA - MA) |
Cancer - Oncology |
2017-12-09
|
emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody) |
hemophilia A |
3 |
Roche (Switzerland) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-09
|
emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody) |
hemophilia A |
3 |
Roche (Switzerland) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-09
|
BMN 270 - valoctocogene roxaparvovec- adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene |
hemophilia A |
1-2 |
Biomarin Pharmaceutical (USA - CA) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-09
|
AMT-061 |
severe and moderately severe hemophilia B |
preclinical |
Uniqure (The Netherlands) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-08
|
Ibrance® (palbociclib) |
postmenopausal women with ER+, HER2- metastatic breast cancer |
3 |
Pfizer (USA - NY) |
Cancer - Oncology |
2017-12-08
|
Ery-asp®/Graspa® (eryaspase - L-asparaginase loaded erythrocytes) |
acute myeloid leukemia |
2b |
Erytech Pharma (France) |
Cancer - Oncology |
2017-12-07
|
CTX001 |
Beta-thalassemia |
1-2 |
Crispr Therapeutics (Switzerland - UK) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-07
|
PCM-075 (1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt) and Zytiga® (abiraterone acetate) |
triple-negative breast cancer (TNBC) |
preclinical |
Trovagene (USA - CA) |
Cancer - Oncology |
2017-12-07
|
emicizumab (ACE910 - anti-factor IXa x anti-factor X humanized bispecific antibody) |
hemophilia A |
3 |
Roche (Switzerland) |
Rare diseases - Genetic diseases - Hematological diseases |
2017-12-07
|
Tedopi® (OSE2101) |
non-small cell lung cancer |
3 |
OSE Pharma (France) now OSE Immunotherapeutics |
Cancer - Oncology |
2017-12-06
|
SPK-8011 |
hemophilia A |
1-2 |
Spark Therapeutics (USA - PA) |
Rare diseases - Genetic diseases - Hematological diseases |