FDA approves Agios Pharmaceuticals’ TIBSOVO for acute myeloid leukemia

The US Food and Drug Administration (FDA) has granted approval to Agios Pharmaceuticals’ TIBSOVO (ivosidenib) for the treatment of adult patients with acute myeloid leukemia (AML) who have relapsed or refractory conditions and are confirmed to have an isocitrate dehydrogenase-1 (IDH1) mutation. This approval marks a significant advancement in targeted cancer therapy, offering new hope to patients with limited treatment options.

TIBSOVO, the first and only FDA-approved therapy for AML patients with an IDH1 mutation, is an oral, targeted inhibitor that disrupts the IDH1 enzyme’s activity. This approval was largely driven by the outcomes of a Phase 1 clinical trial, which involved 174 patients who received a daily dose of 500mg of TIBSOVO until disease progression, the development of unacceptable toxicity, or completion of hematopoietic stem cell transplantation.

The trial demonstrated a combined complete remission (CR) and complete remission with partial hematologic recovery (CRh) rate of 32.8%, with a median duration of CR+CRh of 8.2 months. These results underscore the drug’s effectiveness in achieving strong, durable responses and helping patients maintain transfusion independence.

Hagop M. Kantarjian, professor and chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center, commented on the significance of this approval: “AML patients who relapse or are refractory to available therapies have few, if any, treatment options. The clinical study demonstrated that TIBSOVO has the potential to deliver strong, durable responses as a single agent and can help patients achieve and maintain transfusion independence. IDH inhibitors represent a new class of noncytotoxic, targeted therapies for AML patients with IDH mutations.”

David Schenkein, CEO of Agios Pharmaceuticals, expressed his enthusiasm about the approval: “The FDA approval of TIBSOVO – our first wholly owned drug and the second approved medicine from our research platform in less than a year – is an incredibly exciting milestone for our company and, importantly, for the approximately 6-10% of AML patients with an IDH1 mutation who have been waiting for new treatment options that work radically different than conventional chemotherapy.”

TIBSOVO was approved alongside the Abbott RealTime IDH1 companion diagnostic test, which is essential for identifying eligible patients for treatment with TIBSOVO based on their IDH1 mutation status.