dm+d
Unassigned
New Medicines
High risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myelogenous leukemia (AML)
Information
New molecular entity
Takeda
Takeda
Development and Regulatory status
Discontinued
Discontinued
Discontinued
Oct 21Development discontinued; no longer listed in Takeda pipeline [8].
Category
First-in-class small molecule NEDD8 activating enzyme (NAE inhibitor), tha inhibits the activity of cullin-dependent ubiquitin ligases (CDLs), which are enzyme complexes that control the degradation of proteins involved in cell survival
The incidence of MDS is approximately 4 per 100,000 population a year. It is predominantly a disease of the elderly with an incidence of over 30 per 100,000 a year in those over the age of 70 years [1].
High risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and low-blast acute myelogenous leukemia (AML)
Intravenous infusion
Further information
Yes
Trial or other data
Sep 21PIII PANTHER trial fails to meet primary endpoint. Adding pevonedistat to azacitadine delivered no benefits to event-free survival. PII trials in AML patients who are unfit for intensive chemotherapy are ongoing with regulatory approvals anticipated in 2024 [7].