Pfizer announced that the Food and Drug Administration (FDA) has granted Priority Review for the New Drug Application (NDA) of glasdegib for the treatment of adults with previously untreated acute myeloid leukemia (AML) in combination with low-dose cytarabine (LDAC). The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for December 2018. 

Glasdegib, an investigational oral smoothened (SMO) inhibitor, is a once-daily therapy that is thought to work by disrupting the Hedgehog pathway. Abnormal Hedgehog pathway activation is presumed to be involved in the development of various cancers, including solid tumors and hematologic cancers. 

Gladegib was evaluated in the Phase 2, randomized, open-label, multicenter BRIGHT 1003 study in combination with LDAC vs LDAC alone. The study included 132 patients with previously untreated AML or high-risk myelodysplastic syndrome (MDS) who were not eligible for intensive chemotherapy. 

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The data showed a significant improvement in overall survival (OS), the primary endpoint, with a median 8.8 months in the glasdegib + LDAC group vs 4.9 months in the LDAC alone group. This translated to a 49.9% reduction in mortality risk for patients who received glasdegib + LDAC (hazard ratio [HR] 0.501, 95% CI , 0.334, 0.752; P =.0003). 

Anemia, febrile neutropenia, nausea, decreased appetite, fatigue, and thrombocytopenia were among the most commonly reported side effects in the glasdegib + LDAC group vs LDAC alone. The most common serious adverse events reported were febrile neutropenia and pneumonia.

Glasdegib is also being investigated in BRIGHT AML1019, a Phase 3 trial evaluating the addition of glasdegib to intensive or non-intensive chemotherapy in patients with newly diagnosed AML

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