The US Food and Drug Administration (FDA) has approved Syndax Pharmaceuticals' Revuforj (revumenib) for treating relapsed or refractory (R/R) acute leukaemia with a lysine methyltransferase 2A gene (KMT2A) translocation.
Revuforj is now the first and only menin inhibitor approved for treating patients aged one year and older.
It was expedited through the FDA's Real Time Oncology Review programme, following the grant of breakthrough therapy, fast track designations and priority review to Revuforj.
The therapy’s efficacy was evaluated in the Phase I/II AUGMENT-101 trial, which included 104 patients with the specified acute leukaemia.
In the evaluated group, 21% achieved complete remission with partial haematological recovery. The median duration of these responses was 6.4 months, with a median time of 1.9 months to response. Post-treatment, 23% of patients were able to undergo haematopoietic stem cell transplantation.
The results align with the Phase II interim analysis from the same trial, reinforcing the potential of Revuforj in treating this aggressive leukaemia form.
The safety profile was assessed in 135 patients, forming part of the FDA's analysis for approval.
Syndax Pharmaceuticals CEO Michael Metzger stated: "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 leukaemia’s and mutant NPM1 AML."
The company anticipates the availability of Revuforj 110mg and 160mg tablets through specialty distributors and pharmacies in the US from November 2024.
The 25mg tablets, suitable for patients weighing under 40kg, are expected to be available by early 2025, with an interim oral solution provided through an expanded access programme.
In November 2024 the company entered a synthetic royalty funding agreement with Royalty Pharma, focused on US net sales of Niktimvo (axatilimab-csfr), a treatment for chronic graft-versus-host disease.
