FDA grants breakthrough therapy status to pevonedistat for myelodysplastic syndrome
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The FDA granted breakthrough therapy designation to pevonedistat for patients with higher-risk myelodysplastic syndrome, according to a press release from the drug’s manufacturer.
Pevonedistat (Takeda), a first-in-class NEDD8-activating enzyme inhibitor, demonstrated promising clinical activity combined with azacitidine in a phase 2 study of patients with higher-risk myelodysplastic syndrome, higher-risk chronic myelomonocytic leukemia and acute myeloid leukemia, and in a phase 1 trial of patients with AML.
The agent is currently being evaluated in phase 3 studies as first-line treatment for patients higher-risk myelodysplastic syndrome, higher-risk chronic myelomonocytic leukemia and AML who are ineligible for transplant or intensive induction chemotherapy. Pevonedistat also is being evaluated in combination with azacitidine and venetoclax (Venclexta; AbbVie, Genentech) for AML in a phase 2 study.
“Higher-risk myelodysplastic syndrome is associated with poor prognosis, diminished quality of life and a higher chance of transformation to AML, another aggressive cancer. The combination of pevonedistat and azacitidine is a promising therapeutic approach with the potential to be the first novel treatment advancement for higher-risk myelodysplastic syndrome in more than 10 years,” Christopher Arendt, PhD, head of the oncology therapeutic area unit at Takeda, said in the release. “We thank the FDA for recognizing pevonedistat, and the urgency to develop innovative therapies that address critical treatment needs for higher-risk myelodysplastic syndrome, a patient population with few options.”
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