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Highlights
- SNDX’s Revuforj® included as a category 2A recommendation in NCCN Guidelines for relapsed/refractory NPM1-mutated AML
- Update backed by pivotal AUGMENT-101 trial results, published in Blood (2025)
- Supplemental New Drug Application (sNDA) under FDA Priority Review, with decision expected by October 25, 2025
Syndax Pharmaceuticals, Inc. (Nasdaq:SNDX) announced that the National Comprehensive Cancer Network (NCCN) has updated its Clinical Practice Guidelines in Oncology to include Revuforj® (revumenib) as a recommended treatment option. The inclusion applies to patients with relapsed or refractory acute myeloid leukemia (AML) carrying an NPM1 mutation. Revuforj has been added as a category 2A recommendation, indicating uniform consensus among the NCCN panel that the treatment is an appropriate option based on existing clinical evidence.
The NCCN Guidelines are widely used by oncologists, payers, and healthcare institutions to guide treatment decisions. Inclusion of a therapy often provides greater clinical visibility and may influence prescribing practices across cancer centers in the United States.
Clinical Evidence
The guideline update is supported by pivotal results from the AUGMENT-101 trial, a global, multicenter clinical study that evaluated revumenib in patients with relapsed or refractory NPM1-mutated AML. Data from the study, published in Blood earlier in 2025, demonstrated clinically meaningful outcomes in this difficult-to-treat patient population.
Findings from AUGMENT-101 were also presented at the European Hematology Association (EHA) Annual Congress, further validating the potential of revumenib in this setting. The trial outcomes served as the basis for Syndax’s regulatory submissions and the NCCN’s decision to update its guidelines.
Regulatory Progress
Syndax has filed a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration (FDA) seeking approval of revumenib for NPM1-mutated AML. The FDA accepted the filing under Priority Review and is evaluating it through the Real-Time Oncology Review (RTOR) program, an initiative aimed at accelerating patient access to promising cancer therapies.
A decision on the sNDA is expected by October 25, 2025. If approved, this would expand revumenib’s label to include NPM1-mutated AML, in addition to its existing approval for acute leukemia with KMT2A rearrangements.
Development Pipeline
Revumenib is also being studied in a broader set of clinical programs. Ongoing and planned trials include:
- Evaluation in patients with NPM1 mutations and KMT2A rearrangements across various treatment settings
- Combination approaches with standard chemotherapy or targeted therapies in newly diagnosed AML patients
- Investigations into potential use in earlier lines of treatment and in different AML subtypes
These efforts are intended to establish revumenib as a foundational therapy across multiple genetic subsets of AML.
Industry Context
The NCCN guideline inclusion highlights the growing role of targeted therapies in AML treatment, particularly for molecularly defined subgroups such as NPM1 mutations and KMT2A rearrangements. For Syndax, the development strengthens its position in the hematology space and supports the company’s strategy of advancing targeted oncology medicines.
