FDA Drug Approvals: Oncology and Hematology — Year in Review 2018

Mary L Windle, PharmD


January 15, 2019

New Drugs for Acute Myeloid Leukemia

Xospata (gilteritinib)

Gilteritinib is a small molecule that inhibits multiple tyrosine kinase receptors. It is indicated for patients who have relapsed or refractory acute myeloid leukemia (AML) with an FMS-like tyrosine kinase 3 (FLT3) mutation. Approval was based on interim analysis of the ongoing ADMIRAL clinical trial, a phase 3, open-label, multicenter, randomized study comparing gilteritinib with salvage chemotherapy. Salvage chemotherapy consisted of low-dose cytarabine or azacitidine, or MEC (mitoxantrone, etoposide, and intermediate-dose cytarabine) or FLAG-IDA (fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin). (ClinicalTrials.gov)

Daurismo (glasdegib)

Glasdegib is a hedgehog pathway inhibitor. It is indicated, in combination with low-dose cytarabine, for newly diagnosed acute myeloid leukemia (AML) in adults aged 75 years or older, or adults who have comorbidities that preclude use of intensive induction chemotherapy. Results from the phase 2 BRIGHT 1003 study evaluated glasdegib combined with low-dose cytarabine or cytarabine alone. Median overall survival was 8.8 months for patients treated with glasdegib plus cytarabine compared with 4.9 months for cytarabine alone. This difference represented a nearly 50% reduction in the risk of death for patients treated with glasdegib plus cytarabine. (Leukemia 2018 Dec 16)

Tibsovo (ivosidenib)

Ivosidenib is indicated in patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase type 1 (IDH1) mutation as detected by an FDA-approved test. Approval was based on results of a phase 1 open-label, single-arm, multicenter dose-escalation and expansion trial (n=174). At a median follow-up of 8.3 months, 32.8% (n=57) of patients experienced complete remission (CR) (43 patients) or CR with partial hematologic recovery (14 patients) that lasted for a median of 8.2 months. (N Engl J Med. 2018 Jun 21;378(25):2386-2398)


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