FDA grants approval to glasdegib for the treatment of newly diagnosed AML patients who are ineligible for chemotherapy

On 21 November 2018, the US Food and Drug Administration (FDA) granted approval to glasdegib (Daurismo®),  a once-daily oral medicine, in combination with low-dose cytarabine (LDAC), for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are 75 years or older or who have comorbidities that preclude use of intensive induction chemotherapy. This comes after a priority review was granted to glasdegib in June 2018.

The FDA approval was based on data from the randomized, open-label, phase II BRIGHT 1003 study (NCT01546038), which is evaluating the safety and efficacy of glasdegib (PF-04449913), an oral smoothened inhibitor, with (n = 77) or without (n = 38) LDAC in 115 patients with AML. The results of this study were presented at the 2016 American Society of Hematology (ASH) Annual Meeting by Jorge Cortes from The University Texas MD Anderson Cancer Center, Houston, TX, and reported by the AML Global Portal here.

Results demonstrated a significant improvement in the primary endpoint of overall survival (OS). Median OS was 8.3 months (95% CI, 4.4–12.2) for patients treated with glasdegib plus LDAC compared with 4.3 months (95% CI, 1.9–5.7) for patients treated with LDAC only. This difference represented a 54% reduction in the risk of death for patients treated with glasdegib plus LDAC (HR = 0.46, 95% CI: 0.30–0.71, P = 0.0002) compared to chemotherapy alone.

Jorge Cortes commented on the approval for glasdegib in which he noted that glasdegib plus LDAC reduced the risk of death during the study period by 54 percent compared to chemotherapy alone. This provides a much-needed treatment for those patients for whom intensive chemotherapy is not an option.”