The FDA has approved Daurismo (glasdegib, Pfizer, Inc), a once-daily oral medicine, for the treatment of newly diagnosed AML in adults 75 years of age or older, or adults with comorbidities that preclude intensive induction chemotherapy. It is the first and only FDA-approved Hedgehog pathway inhibitor for AML.
The Hedgehog signaling pathway plays an essential role in the development of the human embryo. In adults, it is thought that abnormal activation of this pathway contributes to the development and persistence of cancer stem cells.
The standard of care for people with AML is intensive chemotherapy, but for many older patients and those with certain health conditions, intensive treatment is not an option. Glasdegib is taken in combination with low-dose cytarabine (LDAC), a type of chemotherapy.
Data from a randomized clinical trial showed a significant improvement in overall survival with glasdegib. Patients treated with glasdegib plus LDAC had a 54 percent reduction in the risk of death compared to those treated with glasdegib alone.
During the trial, the most commonly reported adverse events included anemia, fatigue, hemorrhage, febrile neutropenia, muscle pain, nausea, edema, thrombocytopenia, dyspnea, decreased appetite, dysgeusia, mucositis, constipation, and rash.
Source: Pfizer, Inc, November 21, 2018