Ivosidenib Now FDA-Approved as First-Line Treatment for AML with IDH1 Mutation

FDA Approvals, News & Updates

On May 2, 2019, the US Food and Drug Administration (FDA) approved ivosidenib (Tibsovo; Agios Pharmaceuticals, Inc) for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved companion diagnostic test, in patients who are aged ≥75 years or who have comorbidities that preclude the use of intensive induction chemotherapy.

This is the second FDA approval for ivosidenib, which was previously approved on July 20, 2018, for the treatment of patients with relapsed or refractory AML with a susceptible IDH1 mutation.

The most recent approval of this agent was based on results from the open-label, single-arm, multicenter, clinical trial that included 28 patients with newly diagnosed AML with an IDH1 mutation.

Patients were treated with ivosidenib, which was administered orally at a dose of 500 mg daily until disease progression, development of unacceptable toxicity, or stem-cell transplantation. The primary end point of the trial was the combined complete remission (CR) and complete remission with partial hematologic improvement (CRh) rate.

Twelve (42.9%) of the 28 patients achieved CR+CRh, and 7 (41.2%) of the 17 transfusion-dependent patients achieved transfusion independence lasting ≥8 weeks. Two of the 28 patients underwent stem-cell transplantation following ivosidenib treatment.

Adverse reactions occurring in ≥25% of patients included diarrhea, fatigue, edema, decreased appetite, leukocytosis, nausea, arthralgia, abdominal pain, dyspnea, differentiation syndrome, and myalgia.

The prescribing information for ivosidenib includes a boxed warning that differentiation syndrome may occur and can be fatal if not treated.

Uncategorized - August 7, 2015

Diagnosis and Management of Polycythemia Vera Proceedings from a Multidisciplinary Roundtable

A multidisciplinary roundtable was convened on May 29, 2014, to gain insight and guidance from experts on the diagnosis and management of polycythemia vera (PV), including practical strategies, recent advances, and the emerging science. The roundtable was comprised of 10 experts in relevant fields: hematology, oncology, managed care, specialty pharmacy, [ Read More ]

Lung Cancer, Web Exclusives - May 29, 2019

Personalizing Immunotherapy in NSCLC Using PD-L1 and Tumor Mutation Burden as Biomarkers

Tumor expression of PD-L1 has consistently predicted ­response and survival outcomes in non–small-cell lung cancer (NSCLC), whereas the role of PD-L1 in immune cells is unclear, said ­Edward B. Garon, MD, Director, Thoracic Oncology Program, David Geffen School of Medicine, University of California, Los Angeles, at the 2019 ASCO-SITC Clinical Immuno-Oncology Symposium.