Rigel Pharmaceuticals has secured the US Food and Drug Administration (FDA) approval for Rezlidhia (olutasidenib) to treat a type of acute myeloid leukaemia (AML) in adults.
The drug is indicated for adults with relapsed or refractory (R/R) AML with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, as detected by an FDA-approved test.
Rezlidhia is an oral, small molecule, designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells.
In August 2022, Rigel signed an exclusive, worldwide license agreement with Forma Therapeutics to develop, manufacture and commercialise Rezlidhia.
Under the terms of the agreement, the US drugmaker is responsible for the commercialisation of Rezlidhia in the US, with plans to roll out the product outside the US.
Rigel president and CEO Raul Rodriguez 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 haematology-oncology portfolio.
“I would like to extend our sincerest thanks to all the patients, their families and caregivers, the doctors, the FDA, and our team members who have all contributed to the approval of Rezlidhia.”
The FDA approval is based on data from the open-label Phase 2 registrational study that evaluated Rezlidhia 150mg twice daily dose in 153 mIDH1 R/R AML patients.
In the Phase 2 study, a composite of complete remission (CR) plus complete remission with partial haematological recovery (CRh), was the primary endpoint.
The CRh is defined as less than 5% blasts in the bone marrow, with no evidence of disease, and partial recovery of peripheral blood counts.
Furthermore, Rezlidhia comes with a boxed warning of causing differentiation syndrome, and hepatotoxicity.
Phase 2 trial investigator Jorge E Cortes 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-characterised safety profile.”