FDA D.I.S.C.O. Burst Edition: FDA approval of Rezlidhia (olutasidenib) for relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation
Regulatory approval: On Dec 1, 2022, the FDA approved olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation detected by an FDA-approved test. The approval was supported by Study 2102-HEM-101, an open-label, single-arm, multicenter trial (n=147). Key endpoints included CR+CRh rate (35%) and transfusion independence metrics. Labeling includes a Boxed Warning for potentially fatal differentiation syndrome.
FDA approval (Dec 1, 2022): olutasidenib for IDH1-mutated R/R AML
The FDA granted a regulatory approval on December 1, 2022 for olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test. On the same day, the FDA also approved the Abbott RealTime IDH1 Assay to select patients for olutasidenib.
Approval was based on Study 2102-HEM-101, an open-label, single-arm, multicenter clinical trial enrolling 147 adults with R/R AML and an IDH1 mutation confirmed using the approved assay. Olutasidenib was administered orally at 150 mg twice daily until disease progression, unacceptable toxicity, or hematopoietic stem cell transplantation (HSCT). Median treatment duration was 4.7 months; 16 patients proceeded to HSCT after olutasidenib.
Efficacy was established based on:
- CR + CRh rate: 35% (including 32% CR and **2.7% CRh)
- Median time to CR+CRh: 1.9 months
- Median duration of CR+CRh: 25.9 months
- Transfusion dependence to independence: among 86 transfusion-dependent at baseline, 29 became independent during any 56-day post-baseline period.
The prescribing information includes a Boxed Warning for differentiation syndrome, which can be fatal.