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FDA grants priority review to glasdegib for treatment-naive acute myeloid leukemia
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The FDA granted priority review designation to glasdegib for use with cytarabine for adults with treatment-naive acute myeloid leukemia.
Glasdegib (Pfizer) is an investigational oral agent thought to inhibit the SMO receptor and, thus, disrupting the Hedgehog pathway.
The FDA based the priority review on results from BRIGHT 1003, a randomized phase 2 trial that showed the addition of glasdegib to chemotherapy nearly doubled OS.
“Patients with acute myeloid leukemia who are ineligible for intensive chemotherapy are in critical need of new treatment options to improve their overall survival,” Mace Rothenberg, MD, chief development officer for oncology with Pfizer Global Product Development, said in a company-issued press release. “We are proud that our application was accepted by the FDA for priority review.”
The FDA is expected to make a decision about approval by December.
The open-label, multicenter BRIGHT 1003 trial included 132 patients with previously untreated AML or high-risk myelodysplastic syndrome who were not eligible for intensive chemotherapy. Researchers assigned 88 patients to once-daily glasdegib plus low-dose cytarabine. The other 44 patients received low-dose cytarabine alone.
The results — presented at ASH Annual Meeting and Exposition in 2016 — showed patients assigned glasdegib achieved significantly longer median OS (8.8 months vs. 4.9 months; HR = .5; 95% CI, 0.33-0.75).
Patients assigned glasdegib experienced higher rates of anemia (45% vs. 42%), febrile neutropenia (36% vs 27%), nausea (36% vs 12%), decreased appetite (32% vs 12%), fatigue (31% vs 20%) and thrombocytopenia (30% vs 27%). The most common serious adverse events among glasdegib-treated patients were febrile neutropenia (29% for combination vs 20% for cytarabine alone) and pneumonia (21% vs 17%).
The phase 3 BRIGHT AML 1019 trial — which began enrolling patients earlier this year — will assess the addition of glasdegib to intensive or nonintensive chemotherapy for patients with newly diagnosed AML.