General AML

FDA accepts New Drug Application and grants Priority Review status to glasdegib for the treatment of patients with previously untreated AML

On 27 June 2018, the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) and granted priority review to glasdegib, an oral smoothened (SMO) inhibitor, for the treatment of adult patients with previously untreated acute myeloid leukemia (AML) in combination with low dose chemotherapy (LDAC).

The NDA submission 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) with (n = 88) or without (n = 44) LDAC in 132 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 8.8 months for patients treated with glasdegib plus LDAC compared with 4.9 months for patients treated with LDAC only. This difference represented a 49.9% reduction in the risk of death for patients treated with glasdegib plus LDAC (HR = 0.501, 95% CI: 0.334–0.752, P = 0.0003). In summary, the addition of glasdegib to LDAC for AML and high-risk MDS patients improved OS compared with LDAC alone and this survival benefit was consistent among cytogenetic subgroups.2

At present, glasdegib is being assessed in a phase III BRIGHT AML1019 trial (NCT03416179), which is evaluating the addition of glasdegib to intensive or non-intensive chemotherapy in patients with newly diagnosed AML, began enrolling earlier this year. Jorge Cortes discusses the design of this phase III study here.

  1. Pfizer: U.S. FDA Grants Priority Review for Pfizer’s New Drug Application for Glasdegib in Patients with Previously Untreated Acute Myeloid Leukemia. 2018 June 27. [Accessed 2018 June 28].
  2. Cortes J.E. et al. A Phase 2 Randomized Study of Low Dose Ara-C with or without Glasdegib (PF-04449913) in Untreated Patients with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome. Oral Abstract #99: ASH 58th Annual Meeting and Exposition, San Diego, CA.
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