Takeda's Pevonedistat Receives the US FDA's Breakthrough Therapy Designation to Treat Patients with Higher-Risk Myelodysplastic Syndrome
Shots:
- The BT designation is based on the final analysis of the Pevonedistat-2001 P-II study assessing pevonedistat + azacitidine vs azacitidine as monothx. in patients with rare leukemias- including HR-MDS
- The 1EPs of the study include OS- EFS- CR and transfusion independence- and AE profile. The designation addresses the needs of people living with HR-MDS- for whom few therapies exist- and the benefits are limited
- Pevonedistat is a first in class NEDD8-activating enzyme (NAE) inhibitor. The pre-clinical studies demonstrated the inhibition of NAE- blocked the modification of select proteins- which resulted in the disruption of cell cycle progression and cell survival- leading to cancer cell death
Ref: Takeda | Image: Takeda
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