FDA approves olutasidenib for certain adults with acute myeloid leukemia
The FDA approved olutasidenib for treatment of certain adults with relapsed or refractory acute myeloid leukemia.
The indication applies to use of olutasidenib (Rezlidhia, Rigel Pharmaceuticals) capsules by adults with relapsed or refractory AML with a susceptible IDH1 mutation as detected by an FDA-approved test.
The FDA also approved the Abbott RealTime IDH1 Assay (Abbott Laboratories) to identify patients who are appropriate for olutasidenib treatment.
Olutasidenib is an oral small molecule inhibitor of mutated IDH1 designed to bind to and inhibit mutated IDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells, according to the agent’s manufacturer.
FDA based the approval on results of the single-arm, open-label Study 2102-HEM-101.
The trial included 147 patients with relapsed or refractory AML with an IDH1 mutation.
Patients received 150 mg olutasidenib twice daily until disease progression, unacceptable toxicity or hematopoietic stem cell transplantation.
Efficacy outcomes included rate of complete remission plus complete remission with partial hematologic recovery, duration of complete remission plus complete remission with partial hematologic recovery, and rate of conversion from transfusion dependence to transfusion independence.
Results showed 35% (95% CI, 27-43) of patients achieved complete remission (32%) or complete remission with partial hematologic recovery (2.7%).
Researchers reported median time to complete remission or complete remission with partial hematologic recovery of 1.9 months (range, 0.9-5.6), with a median duration of 25.9 months (95% CI, 13.5-not reached).
"We are delighted by the approval of Rezlidhia based on the strength of data supporting the efficacy and safety of the product," Raul Rodriguez, president and CEO of Rigel Pharmaceuticals, said in a company press release. "Rezlidhia provides a new and important, oral therapy option for patients who typically have a poor clinical outcome.”
Adverse events that occurred among at least 20% of olutasidenib-treated patients included nausea, fatigue/malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea and transaminitis. The prescribing information contains a boxed warning about the risk for differentiation syndrome.
References:
- FDA. FDA approves olutasidenib for relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation (press release). Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-olutasidenib-relapsed-or-refractory-acute-myeloid-leukemia-susceptible-idh1-mutation. Published Dec. 1, 2022. Accessed Dec. 1, 2022.
- Rigel Pharmaceuticals. Rigel announces U.S. FDA approval of Rezlidhia (olutasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation (press release). Available at: https://www.rigel.com/investors/news-events/press-releases/detail/349/rigel-announces-u-s-fda-approval-of-rezlidhia. Published Dec. 1, 2022. Accessed Dec. 1, 2022.
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