Rigel Pharmaceuticals gets Rezlidhia FDA approval for AML

Rigel Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its Rezlidhia (olutasidenib) capsules for relapsed or refractory (R/R) acute myeloid leukemia (AML) in adult patients.

Rezlidhia is indicated for patients with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as identified by an FDA-approved test.

The oral, small molecule and mutant isocitrate dehydrogenase-1 (mIDH1) inhibitor is engineered to bind and prevent mIDH1 to minimize 2-hydroxyglutarate levels and for restoring normal cellular differentiation of myeloid cells.

Rezlidhia FDA approval was based on a phase 2 study that showed 35% of 147 patients had complete remission or complete remission with partial recovery in mIDH1 R/R AML patients with a median duration of response of 25.9 months.

Jorge E. Cortes — Georgia Cancer Center Director and Cecil F Whitaker Jr and the phase 2 trial investigator, commenting on Rezlidhia FDA approval, said: “Rezlidhia is a novel, non-intensive monotherapy treatment in the relapsed/refractory AML setting demonstrating a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission.

“The 25.9 months median duration of CR+CRh is a clinically meaningful improvement for AML patients and appears to be longer than currently available treatment options.

“Given the limited treatment options for adult patients with mIDH1 R/R AML, who typically have a poor prognosis, REZLIDHIA may provide an effective, new treatment option with a well characterized safety profile.”

In August 2022, Rigel Pharmaceuticals acquired the rights to develop, produce and commercialize Rezlidhia from Novo Nordisk-owned Forma Therapeutics for an upfront payment of $2 million with additional payments on achievement of certain milestones.

The Nasdaq-listed biotechnology company will launch and commercialize the new AML drug in the US and plans to work with potential partners to further develop and commercialize the product outside the country as part of the license agreement.

Commenting on Rezlidhia FDA approval, Raul Rodriguez — Rigel Pharmaceuticals president and CEO, said: “We are delighted by the approval of REZLIDHIA based on the strength of data supporting the efficacy and safety of the product.

“REZLIDHIA provides a new and important, oral therapy option for patients who typically have a poor clinical outcome. Additionally, this approval greatly strengthens and expands Rigel’s commercial hematology-oncology portfolio.”