FDA Approves Revuforj to Treat R/R Acute Leukemia with KMT2A Translocation

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FDA approved Revuforj (revumenib) to treat relapsed or refractory acute leukemia with a lysine methyltransferase 2a gene tanslocation in adults and pediatric patients at least one year old.¹ The medication, developed by Syndax is a menin inhibitor and is the first of its kind to be approved by FDA.

Approval for the drug came based on the results of the AUGMENT-101 clinical trial, where it showed strong results in treating patients. According to Syndax, 110 and 160 mg tablets of Revuforj should start being available through specialty distributors in November.

In a press release, Syndax CEO Michael A. Metzger said, “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."

In the same press release, Ghayas C. Issa, MD, associate professor of Leukemia at The University of Texas MD Anderson Cancer Center 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."

In July of this year, Syndax announced that the prescription drug user fee act for the new drug application for revmenib had been extended to December of this year.²

At the time, Metzger said, “Revumenib, upon approval, will be the first drug indicated to treat patients with KMT2A-rearranged acute leukemia, a population with significant unmet need. We are confident that the data from the AUGMENT-101 trial, as well as the additional information provided to the FDA, support approval and continue to demonstrate the meaningful benefit revumenib brings to patients with this devastating disease. We look forward to continuing our engagement with the FDA as they complete their review of the NDA by December 26, 2024."

Revuforj is just one of the medications in Syndax’s pipeline. Earlier this month, the company announced that it had entered into a $350 million royalty funding agreement for Niktimvo with Royalty Pharma.³

At the time, Metzger said, “We expect this transaction to fund us through profitability, while ensuring that we continue to participate in the profits from Niktimvo and retain the upside of its future growth. With this significant infusion of capital, we are well positioned to successfully launch two first-in-class medicines and expand their opportunity with additional indications. Royalty Pharma shares our belief that Niktimvo can create significant value as a new treatment option for patients with chronic graft-versus-host disease (GVHD) and recognizes its multi-billion-dollar franchise potential."

In the same press release, Royalty Pharma CEO and founder Pablo Legorreta said, “We are excited to partner with Syndax, an innovative oncology company with an exciting pipeline. Niktimvo is a first-in-class product that has the potential to address the serious and devastating complications associated with chronic GVHD, where there is clear unmet need for additional treatment options. We look forward to Syndax and their partner Incyte launching Niktimvo soon and bringing this important medicine to GVHD patients."

Sources

1. Syndax Announces FDA Approval of Revuforj® (revumenib), the First and Only Menin Inhibitor to Treat Adult and Pediatric Patients with Relapsed or Refractory Acute Leukemia with a KMT2A Translocation. Syndax. November 15, 2024. https://ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-first-and-only
2. Syndax Announces PDUFA Action Date Extension for Revumenib NDA for Relapsed or Refractory KMT2Ar Acute Leukemia. Syndax. July 29, 2024. Accessed November 18, 2024. https://ir.syndax.com/news-releases/news-release-details/syndax-announces-pdufa-action-date-extension-revumenib-nda
3. Syndax Pharmaceuticals and Royalty Pharma Enter into $350 Million Royalty Funding Agreement for Niktimvo. Syndax. November 4, 2024. Accessed November 18, 2024. https://ir.syndax.com/news-releases/news-release-details/syndax-pharmaceuticals-and-royalty-pharma-enter-350-million