Glasdegib for adult patients with leukemiaFebruary 5, 2019
The USFDA approved glasdegib (Daurismo), a once-daily oral medicine, for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are 75 years or older.
Glasdegib is taken in combination with low-dose cytarabine (LDAC), a type of chemotherapy.
Pfizer said glasdegib is the first and only FDA-approved Hedgehog pathway inhibitor for AML. The Hedgehog signalling pathway plays an essential role in embryogenesis.
In the pivotal, randomized, international Phase 2 BRIGHT 1003 trial, 115 patients with newly diagnosed AML were randomized 2:1 to receive glasdegib plus LDAC or LDAC alone. Of the 77 patients treated with glasdegib plus LDAC, more than half had secondary AML, or AML that develops as a result of prior blood/bone marrow conditions or previous anticancer therapy. Eleven of the 39 patients with secondary AML received prior treatment with a hypomethylating agent. Historically, the prognosis is poor for these patients and treatment options have been limited to clinical trials or palliative care.