FDA Approves First Targeted Treatment for Relapsed or Refractory AML

The FDA cleared Agios Pharmaceuticals’ Tibsovo (ivosidenib) as the first targeted treatment for patients with relapsed or refractory acute myeloid leukemia with a certain mutation.

The treatment is approved for use with a companion Abbott Laboratories diagnostic to detect specific mutations in the IDH1 gene. The agency based the approval on a single-arm, 174-patient trial.

The drug must be dispensed with a patient medication guide and will include a boxed warning about the potential for an adverse reaction known as differentiation syndrome.