The steady pace of progress in treating patients with acute myeloid leukemia (AML), still one of the most deadly blood cancers, continued with today’s U.S. Food & Drug Administration’s second approval of a drug called ivosidenib (Tibsovo) that works for patients with a specific subtype of AML.
In another win for precision medicine, where therapies are matched to a patient’s specific genetic profile, the FDA approved ivosidenib for newly diagnosed patients 75-years-of-age and older with a genetic mutation known as IDH1. The FDA had approved ivosidenib in June 2018 for patients who had relapsed or did not respond to previous therapy.
While today’s approval of ivosidenib is based on a clinical trial in which newly diagnosed patients were treated with ivosidenib as a single agent, the drug continues to be tested in other trials, including LLS’s Beat AML Master Clinical trial, as a first-line therapy in combination with the chemotherapy drug azacitidine.
In 2016, LLS launched the groundbreaking Beat AML precision medicine study to test multiple targeted therapies simultaneously at multiple cancer centers around the country. Ivosidenib is being tested in Beat AML for newly diagnosed patients who are 60-years- of-age and older.
Today’s approval is based on findings from a phase 1 trial in which ivosidenib induced a 28.6% complete response (CR) rate and a CR plus CR with partial hematologic recovery (CRh) rate of 42.9%.
“For too long we’ve treated AML as a one-size-fits-all disease where it is really multiple subtypes of disease, and the outcomes for patients have been dismal,” said Louis J. DeGennaro, Ph.D., president and CEO of LLS. “Now, after four decades and millions of dollars invested, we are finally seeing results for AML patients that give us real reason for optimism.”