Novel Drug Granted Priority Review for Acute Myeloid Leukemia Treatment
The Food and Drug Administration (FDA) has granted a priority review to a new drug application (NDA) for glasdegib to treat patients with previously untreated acute myeloid leukemia (AML) in combination with low-dose cytarabine (LDAC), which is a type of chemotherapy, according to Pfizer, the manufacturer of the drug.
The priority review of glasdegib, an investigational oral smoothened (SMO) inhibitor, is based on the randomized, open-label, multicenter phase 2 BRIGHT 1003 study, which compared glasdegib plus LDAC to LDAC alone in 132 patients with previously untreated AML or high-risk myelodysplastic syndrome (MDS) who were not eligible to receive intensive chemotherapy.
Patients in the combination arm experienced longer overall survival (OS) compared to those who only had LDAC (8.8 months vs. 4.9 months) – a near 50 percent reduction in the risk of death.
“Glasdegib is the first smoothened inhibitor to potentially offer such a benefit to patients with acute myeloid leukemia, and we are proud that our application was accepted by the FDA for priority review,” Mace Rothenberg, M.D., chief development officer of oncology at Pfizer Global Product Development, said in a statement.
The most frequently reported side effects occurred in more patients in the combination arm compared with the control arm, which included anemia (45 percent vs. 20 percent), febrile neutropenia (36 percent vs. 27 percent), nausea (36 percent vs. 12 percent), decreased appetite (32 percent vs. 12 percent), fatigue (31 percent vs. 20 percent) and thrombocytopenia (30 percent vs. 27 percent).
Similarly, serious side effects occurred in more patients in the combination arm, which included febrile neutropenia (29 percent vs. 20 percent) and pneumonia (21 percent vs. 17 percent).
The FDA is set to make its decision in December 2018.
“Patients with acute myeloid leukemia who are ineligible for intensive chemotherapy are in critical need of new treatment options to improve their overall survival,” Rothenberg said.