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].

Evidence based evaluations