EU approves Daurismo for acute myeloid leukemia.- Pfizer

Pfizer announced that the European Commission approved Daurismo (glasdegib), a Hedgehog pathway inhibitor, in combination with low-dose cytarabine (LDAC), a type of chemotherapy, for the treatment of newly diagnosed (de novo or secondary) acute myeloid leukemia (AML) in adult patients who are not candidates for standard chemotherapy. The approval follows the medicine’s positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) earlier this year, as well as the medicine’s approval by the U.S. Food and Drug Administration (FDA) in November 2018.The European Commission’s approval of Daurismo is based on results from the Phase II BRIGHT 1003 trial, which showed Daurismo nearly doubled median overall survival compared to LDAC alone (8.3 months vs. 4.3 months, HR 0.463, 95% CI [0.299,0.717]) in patients with previously untreated (de novo or secondary) AML who were not eligible for intensive chemotherapy. The difference represented a 54 percent reduction in the risk of death for patients treated with Daurismo plus LDAC (HR: 0.463, 95% CI: 0.299, 0.717, one-sided p-value 0.0002). In the Phase II BRIGHT 1003 trial, 116 patients with previously untreated de novo or secondary AML who were not eligible to receive intensive chemotherapy were randomized 2:1 to receive Daurismo plus LDAC or LDAC alone. Of the 78 patients treated with Daurismo plus LDAC, more than half (51%, 40 patients) had secondary AML, or AML that develops as a result of prior blood/bone marrow conditions or previous anticancer therapy. Eleven of the 40 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. The most frequently (at least 20%) reported adverse reactions in patients receiving Daurismo were anemia (45.2%), hemorrhages (45.2%), febrile neutropenia (35.7%), nausea (35.7%), decreased appetite (33.3%), fatigue (30.9%) and muscle spasms (30.9%).