Syndax Pharmaceuticals has gained FDA approval for Revuforj to treat relapsed/refractory (R/R) acute leukaemia.
The oral dosage form is suitable for both adult and paediatric patients with a KMT2A translocation.
Owing to the unique nature of Revuforj, the FDA previously granted the drug Breakthrough Therapy and Fast Track Designation, as well as Priority Review.
The FDA's decision was based on its analysis of 104 patients with this subset of leukaemia who were treated with Revuforj in the Phase I/II AUGMENT-101 trial.
During the clinical study, the complete remission (CR) rate was 21%, while the median CR duration was 6.4 months.
Of those enrolled, dose reduction and permanent discontinuation was necessary in 10 and 12% of patients, respectively.
Syndax believes that 110 and 160mg tablets will be available in specialty pharmacies across the US by November.
The 25mg tablets, which are recommended for use in patients under 40kg, will be available between the first and second quarter of 2025.
Addressing unmet needs
KMT2A gene rearrangements are responsible for an aggressive form of acute leukaemia which has a poor prognosis and high relapse rate.
More than 95% of patients with KMT2Ar acute leukaemia are estimated to have a KMT2A tranlocation, and more than half of these patients will relapse after receiving frontline therapies.
Currently, the median overall survival (OS) of patients with this indication is less than one year.
Therefore, the approval of Revoforj could offer patients with R/R acute leukaemia new hope, explains CEO of Syndax Michael Metzger:
"The approval of Revuforj is a remarkable achievement that reflects the dedication and tenacity of everyone involved, especially the patients and clinicians who participated in our trial and our talented Syndax team,"
"We are well-prepared to launch Revuforj this month and we are committed to rapidly advancing the development of Revuforj across the treatment continuum for KMT2A-rearranged acute leukemias and mutant NPM1 AML."
Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center, Ghayas Issa, added: "The FDA approval of the first menin inhibitor is a major breakthrough for patients with R/R acute leukemia with a KMT2A translocation, a genetic alteration associated with a very poor prognosis,"
"The significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients."