FDA approves Tibsovo to treat AML with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation.- Agios Pharma.

Agios Pharmaceuticals, Inc. has announced that Tibsovo (ivosidenib) was granted approval from the FDA for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA approved test. Tibsovo, an oral, targeted inhibitor of the IDH1 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH1 mutation.

AML is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with approximately 20,000 new cases estimated in the U.S. each year. The majority of patients with AML eventually relapse. Relapsed or refractory AML has a poor prognosis. The five-year survival rate is approximately 27%.3 For 6 to 10 percent of AML patients, the mutated IDH1 enzyme blocks normal blood stem cell differentiation, contributing to the genesis of acute leukemia.