FDA Approves CytoCell CDx for Revumenib in KMT2A-Mutant Acute Leukemia

The FDA has approved the CytoCell KMT2A Breakapart FISH Probe Kit PDx as a companion diagnostic (CDx) to accompany revumenib (Revuforj), a first-in-class Menin inhibitor for the treatment of relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients 1 year and older.¹

The CytoCell PDx detects clinically relevant rearrangements in patients with acute leukemia to identify if they are eligible for treatment with revumenib in a fast, accessible manner.

“We are delighted that our CytoCell KMT2A Breakapart FISH Probe Kit PDx has received marketing authorization as a CDx for Revuforj,” said Leila Luheshi, MD, vice president of Pharma Partnering at OGT, in a press release. “The development and subsequent authorization of this new CDx is an important demonstration of the skill and commitment of our clinical scientists and regulatory specialists to deliver safe and effective diagnostics for patients with one of the most devastating forms of leukemia.”

What Are the Approved Indications for Revumenib?

The FDA approved revumenib in relapsed/refractory KMT2A-rearranged acute leukemia in November 2024.² Data from the phase 1/2 AUGMENT-101 trial (NCT04065399), as well as the additional information provided to the FDA, supported this approval.

In AUGMENT-101, revumenib led to complete remissions (CRs) with a CR with partial hematologic recovery (CRh) rate of 21.2% (95% CI, 13.8%–30.3%) among adult and pediatric patients with relapsed/refractory acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) harboring KMT2A rearrangements, meeting the study’s primary end point.^2,3 The median CR with CRh duration was 6.4 months (95% CI, 2.7–not estimable).

In June 2025, the FDA granted priority review to the supplemental new drug application (sNDA) of revumenib for the treatment of relapsed/refractory NPM1-mutant AML.⁵ This sNDA is also supported by the positive pivotal data from the AUGMENT-101 trial. Results from the NMP1 study cohort were presented at the European Hematology Association (EHA) Annual Congress Meeting in June 2025. These data showed a CR+CRh rate of 26% (n = 20/77; 95% CI, 17%–37%). The median duration of CR/CRh response was 4.7 months (95% CI, 2.1–8.2), and the median time to first CR/CRh was 2.8 months (range, 0.9–8.8). MRD status was assessed in 19 of 20 patients who achieved CR/CRh, 63% (n = 12) of whom were negative for MRD.^3,4

REFERENCES:

1. U.S. Food and Drug Administration authorises CytoCell KMT2A Breakapart FISH Probe Kit PDx as a new companion diagnostic for KMT2A rearranged acute leukaemia. News release. OGT. September 22, 2025. Accessed September 22, 2025. https://tinyurl.com/5ax5ntx4

2. FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation. News release. FDA. November 15, 2024. Accessed September 22, 2025. https://tinyurl.com/mryw44wf

3. Issa GC, Aldoss I, Thirman MJ, et al. Menin inhibition with revumenib for KMT2A-rearranged relapsed or refractory acute leukemia (AUGMENT-101). J Clin Oncol. Published online August 9, 2024. doi:10.1200/JCO.24.00826

4. Syndax presents positive data from pivotal AUGMENT-101 trial of revumenib in relapsed/refractory KMT2Ar acute leukemia at late-breaking oral presentation during 65th ASH Annual Meeting. News release. Syndax. December 12, 2023. Accessed August 16, 2024. ​​https://tinyurl.com/2p3yzrez

5. Syndax announces FDA priority review of sNDA for Revuforj® (revumenib) in relapsed or refractory mNPM1 acute myeloid leukemia. News release. Syndax Pharmaceuticals. June 24, 2025. Accessed September 22, 2025. https://tinyurl.com/nnfc84sh