May 2, 2019 (Global Newswire) — Cambridge, MA
Agios Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) to update the U.S. prescribing information for Ivosidenib. This IDH1 (isocitrate dehydrogenase-1) inhibitor is now indicated for newly diagnosed adult patients with acute myeloid leukemia (AML) who have a confirmed susceptible IDH1 mutation—as detected by an FDA-approved test—and who are either aged 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy.
This application was granted Priority Review designation and was included in the FDA’s "Real-Time Oncology Review" pilot program, an initiative designed to enhance the efficiency of oncology drug reviews by facilitating early access to clinical trial data. Ivosidenib was first approved in July 2018 for use in adult patients with relapsed or refractory (R/R) IDH1-mutated AML. "Although several new AML drugs have been approved over the past two years, many newly diagnosed patients remain ineligible for existing therapies or combination regimens due to their age and comorbidities," stated Chris Bowden, MD, Chief Medical Officer at Agios. "This label expansion for Ivosidenib enables us to provide a targeted oral therapy to patients with an *IDH1* mutation who have no other treatment options. Concurrently, we are exploring the efficacy of Ivosidenib in combination with intensive chemotherapy and azacitidine in newly diagnosed AML patients through two Phase 3 clinical trials. We extend our sincere gratitude to the patients, nurses, physicians, and caregivers who participated in these clinical trials, as well as to the entire Agios team for their exceptional contributions."
"Phase 1 trials of Ivosidenib confirmed that this oral monotherapy can induce durable remissions in newly diagnosed AML patients with an *IDH1* mutation," noted Gail J. Roboz, MD, Professor of Medicine, Director of the Leukemia Program, and Member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. "Many patients enrolled in the study presented with characteristics of aggressive AML subtypes, including secondary disease, high-risk genetic alterations, and prior treatment with hypomethylating agents."
Ivosidenib Overview
Ivosidenib is indicated for adult patients with acute myeloid leukemia (AML) who have a susceptible *IDH1* mutation as detected by an FDA-approved test:
Newly diagnosed AML patients who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy;
Patients with relapsed or refractory AML.
