FDA approves first targeted therapy for myelodysplastic syndromes

October 22, 2023

The U.S. Food and Drug Administration has granted approval for Tibsovo (ivosidenib) as a treatment for adult patients with relapsed or refractory (R/R) myelodysplastic syndromes (MDS) characterized by an isocitrate dehydrogenase-1 (IDH1) mutation, confirmed through an FDA-approved test. 

This marks a significant milestone as the first targeted therapy for this specific condition. The Abbott RealTime IDH1 Assay has also received approval as a companion diagnostic for the identification of R/R MDS patients with an IDH1 mutation. 

MDS is a rare blood cancer condition caused by mutations in bone marrow progenitor cells, resulting in insufficient healthy blood cells. Approximately 60,000 to 170,000 individuals in the U.S. are living with MDS, with an annual global incidence of 87,000 new cases.

Tibsovo had previously gained approval for different indications, including newly-diagnosed Acute Myeloid Leukemia, relapsed or refractory AML, and locally advanced or metastatic cholangiocarcinoma. The Abbott RealTime IDH1 Assay was also previously approved as a companion diagnostic for identifying AML patients with an IDH1 mutation for treatment with Tibsovo or Rezlidhia.