This section highlights FDA-related milestones and regulatory updates for drugs developed by Syndax Pharmaceuticals (SNDX).
Over the past two years, Syndax Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Axatilimab and Revumenib. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Axatilimab - FDA Regulatory Timeline and Events
Axatilimab is a drug developed by Syndax Pharmaceuticals for the following indication: Chronic Graft Versus Host Disease (cGVHD).
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Axatilimab
- Announced Date:
- August 14, 2024
- Indication:
- Chronic Graft Versus Host Disease (cGVHD)
Announcement
Syndax Pharmaceuticals and Incyte announced that the U.S. Food and Drug Administration (FDA) has approved Niktimvo™ (axatilimab-csfr), an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs.).
AI Summary
Syndax Pharmaceuticals and Incyte announced that the FDA has approved Niktimvo™ (axatilimab‑csfr), the first anti-CSF-1R antibody used to treat chronic graft-versus-host disease (GVHD). This approval is for adult and pediatric patients who weigh at least 40 kg (88.2 lbs.) and have not responded to at least two prior lines of systemic therapy.
The FDA based its decision on data from the AGAVE‑201 study, which showed durable responses in multiple organs and patient subgroups. Niktimvo works by targeting key factors that drive inflammation and fibrosis, offering a new treatment option for patients with advanced chronic GVHD. This novel mechanism of action provides hope for those whose disease has progressed despite previous therapies, marking a significant advancement in addressing the serious complications associated with chronic GVHD.
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Revumenib - FDA Regulatory Timeline and Events
Revumenib is a drug developed by Syndax Pharmaceuticals for the following indication: Relapsed/Refractory KMT2Ar Acute Leukemia.
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Revumenib
- Announced Date:
- June 24, 2025
- Estimated Event Date Range:
- October 25, 2025 - October 25, 2025
- Target Action Date:
- October 25, 2025
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that PDUFA action date set for October 25, 2025
AI Summary
Syndax Pharmaceuticals recently announced that the U.S. FDA has granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj® (revumenib). The review, conducted under the Real-Time Oncology Review (RTOR) program, carries a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. This decision marks a significant step as revumenib, an oral menin inhibitor, could become the first and only treatment approved for both relapsed or refractory (R/R) mutant NPM1 acute myeloid leukemia (AML) and R/R KMT2A-rearranged acute leukemia.
The RTOR program allows the FDA to work closely with Syndax to enhance review efficiency and provide early feedback. If approved, revumenib could offer a new treatment option for patients with limited alternatives, reinforcing Syndax’s leadership in developing innovative cancer therapies.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- June 24, 2025
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj® (revumenib) for the treatment of relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML).
AI Summary
Syndax Pharmaceuticals announced that the FDA has granted Priority Review for its supplemental New Drug Application (sNDA) for Revuforj® (revumenib) for treating relapsed or refractory (R/R) mutant NPM1 acute myeloid leukemia (AML). The sNDA is under review via the FDA’s Real-Time Oncology Review (RTOR) program, and the agency has set a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. Revuforj, an oral, first-in-class menin inhibitor, is supported by compelling data from the AUGMENT-101 trial, which demonstrated its potential to effectively treat patients with mNPM1 AML. If approved, Revuforj would offer a new and targeted treatment option for a patient group with limited alternatives, potentially becoming the first and only menin inhibitor approved for this indication.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- June 12, 2025
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced new data from the pivotal AUGMENT-101 trial of Revuforj® (revumenib), the Company's first-in-class menin inhibitor, in patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) and NUP98-rearranged (NUP98r) acute myeloid leukemia (AML).
AI Summary
Syndax Pharmaceuticals announced promising new data from the pivotal AUGMENT-101 trial for Revuforj® (revumenib), their first-in-class menin inhibitor, in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) harboring mutant NPM1 and NUP98 rearrangements. In the Phase 2 cohort of R/R mNPM1 AML patients, the trial showed a 26% complete remission plus partial recovery (CR+CRh) rate and a 48% overall response rate, with robust responses observed regardless of co-mutations, previous therapies, or prior venetoclax exposure. Additionally, a sub-analysis revealed a promising median overall survival of 23 months among responders. In the Phase 1 arm for patients with R/R NUP98r AML, a 60% overall response rate was reported. These results underscore revumenib’s potential to become a standard treatment option for AML patients with these specific genetic alterations.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- December 9, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced additional positive data from the AUGMENT-101 trial of Revuforj® (revumenib) in relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) and the BEAT AML trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed AML patients.
AI Summary
Syndax Pharmaceuticals announced promising results from two key AML studies evaluating Revuforj® (revumenib). In the AUGMENT-101 trial for relapsed or refractory mutant NPM1 AML, data from a Phase 2 cohort showed a 26% rate of complete remission (including partial hematologic recovery) and a 48% overall response rate among evaluable patients. These responses were seen across major subgroups, including those with multiple prior therapies, suggesting that revumenib may offer new hope for difficult-to-treat patients.
Additionally, the BEAT AML trial evaluated revumenib in combination with venetoclax and azacitidine in newly diagnosed AML patients. In this study, all 37 efficacy-evaluable patients achieved an overall response, with a composite complete remission rate of 95%. The high response rates and safety profile of the combination support the potential for revumenib to improve frontline treatment options for AML patients.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- December 7, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals presented positive data from multiple trials of Revuforj® (revumenib) as a single-agent and in combination with standard of care agents in patients with acute leukemias in oral sessions at the 66th American Society of Hematology (ASH) Annual Meeting being held in San Diego, December 7-10, 2024.
AI Summary
Syndax Pharmaceuticals presented promising clinical data for Revuforj® (revumenib) at the 66th American Society of Hematology Annual Meeting in San Diego, December 7–10, 2024. The data included multiple trials assessing Revuforj as both a single agent and in combination with standard treatments in patients with acute leukemias. Notably, in the Phase 1/2 SAVE trial for relapsed/refractory AML, the combination regimen of Revuforj with venetoclax and decitabine/cedazuridine achieved an 82% overall response rate and a 48% complete remission/complete remission with partial hematologic recovery rate.
Additional findings from the Phase 2 AUGMENT-101 trial in patients with R/R KMT2A-rearranged acute leukemia showed rapid and durable responses across all major patient subgroups, high rates of minimal residual disease negativity, and successful transitions to hematopoietic stem cell transplant. These encouraging results support further development and future combination trials, including studies in the frontline therapy setting.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- November 15, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Revuforj® (revumenib) as the first and only menin inhibitor for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.
AI Summary
Syndax Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has approved Revuforj® (revumenib) as the first and only menin inhibitor for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation. This approval is significant because Revuforj is now available for both adult and pediatric patients aged one year and older who are facing this aggressive form of leukemia.
The FDA’s decision was based on strong data from the AUGMENT-101 clinical trial, which demonstrated that Revuforj delivered robust and durable rates of remission in patients. This breakthrough in precision medicine provides a much-needed treatment option for those with limited alternatives, offering new hope for improved outcomes in this high-risk patient population.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- August 12, 2024
- Estimated Event Date Range:
- December 26, 2024 - December 26, 2024
- Target Action Date:
- December 26, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that NDA filing for revumenib in R/R KMT2Ar acute leukemia is being reviewed under RTOR; PDUFA action date of December 26, 2024 –
AI Summary
Syndax Pharmaceuticals announced that its new drug application (NDA) for revumenib is now under review by the FDA through the Real-Time Oncology Review (RTOR) program. Revumenib is a first-in-class menin inhibitor aimed at treating adult and pediatric patients with relapsed or refractory KMT2A-rearranged acute leukemia. The company highlighted that the pivotal AUGMENT-101 trial met its primary endpoint, providing encouraging results that helped drive the NDA submission.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of December 26, 2024, signaling a significant step toward a potential approval. This milestone builds on the positive data published in the Journal of Clinical Oncology, underscoring revumenib's promising clinical profile and marking an important advance in treatment options for patients with limited alternatives.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- August 12, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib, a first-in-class menin inhibitor, in adult and pediatric patients with relapsed/refractory (R/R) KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL) have been published in the Journal of Clinical Oncology.
AI Summary
Syndax Pharmaceuticals recently announced that the pivotal Phase 2 data from the AUGMENT-101 trial have been published in the Journal of Clinical Oncology. The study focused on revumenib, a first-in-class menin inhibitor, in both adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL). The trial met its primary endpoint during the interim analysis, showing a complete remission (CR) or CR with partial hematologic recovery (CRh) rate of 23% among evaluable patients. These promising results highlight revumenib’s consistent and meaningful clinical activity in a patient population that has had limited treatment options. The published data underline the potential of revumenib to serve as a paradigm-changing therapy, supporting its continued development and offering renewed hope for patients battling these aggressive forms of leukemia.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- July 29, 2024
- Estimated Event Date Range:
- September 26, 2024 - September 26, 2024
- Target Action Date:
- September 26, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) for revumenib for the treatment of adults and pediatric patients with relapsed or refractory (R/R) KMT2Ar acute leukemia.
AI Summary
Syndax Pharmaceuticals announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) action date for its New Drug Application (NDA) review of revumenib. This extension, now set for December 26, 2024, gives the agency additional time to thoroughly evaluate the supplemental information provided by the company. The additional review time was needed after the FDA determined that the new data constituted a Major Amendment to the NDA, thereby triggering a standard three‐month extension from the initial deadline of September 26, 2024.
Revumenib is being developed to treat both adults and pediatric patients with relapsed or refractory KMT2Ar acute leukemia—a group that has significant unmet medical needs. The extra time will allow the FDA to continue its detailed review of data supporting revumenib’s potential benefits in addressing this serious condition.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- June 14, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced updated data from multiple combination trials of revumenib, the Company's potent, selective, small molecule menin inhibitor, in patients with acute leukemias.
AI Summary
Syndax Pharmaceuticals updated data from several combination trials of revumenib, its potent, selective menin inhibitor, in patients with acute leukemias. In the BEAT AML trial, revumenib was tested alongside venetoclax and azacitidine in older patients with newly diagnosed mutant nucleophosmin (mNPM1) or KMT2A-rearranged AML. The interim results showed an impressive 96% composite complete remission rate, with most patients also achieving minimal residual disease-negative status. In another study, the AUGMENT-102 trial evaluated revumenib in combination with fludarabine-cytarabine in relapsed or refractory acute leukemia patients, where a 52% composite complete remission rate was observed. These trials highlight revumenib’s potential to enhance standard treatment regimens while maintaining a manageable safety profile. Syndax plans to advance revumenib into further pivotal trials, supporting its promise as a key treatment option in various acute leukemia settings.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- June 6, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced that it has advanced into the Phase 1b portion of its Phase 1/2 proof-of-concept trial of revumenib, the Company's highly selective, oral menin inhibitor, as a monotherapy in patients with relapsed or refractory (R/R) metastatic microsatellite stable (MSS) colorectal cancer (CRC).
AI Summary
Syndax Pharmaceuticals announced an advancement into the Phase 1b portion of its Phase 1/2 proof-of-concept trial for revumenib, a highly selective oral menin inhibitor. The study, which focuses on patients with relapsed or refractory metastatic microsatellite stable colorectal cancer (MSS CRC), has moved forward after a favorable review by the Independent Data Monitoring Committee. Initial data from the earlier Phase 1a portion showed that revumenib was well tolerated, with no Grade 3 or higher treatment-related adverse events observed. In addition, early signs of efficacy were noted, including a 33% stable disease rate at 16 weeks, which compared favorably to current standard treatments. This advancement into Phase 1b further explores revumenib as a monotherapy and marks a significant step in expanding its potential use beyond hematological cancers.
Read Announcement- Drug:
- Revumenib
- Announced Date:
- April 8, 2024
- Indication:
- Relapsed/Refractory KMT2Ar Acute Leukemia
Announcement
Syndax Pharmaceuticals announced the presentation of positive data from the pivotal AUGMENT-101 trial in pediatric patients with relapsed/refractory (R/R) KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL) treated with revumenib, a first-in-class menin inhibitor.
AI Summary
Syndax Pharmaceuticals has announced positive results from the pivotal AUGMENT-101 trial in pediatric patients with relapsed/refractory KMT2A-rearranged acute leukemia, including both AML and ALL. In this trial, revumenib—a first-in-class menin inhibitor—demonstrated promising activity with a consistent safety profile, similar to that observed in adult populations. Importantly, a significant number of pediatric patients who achieved an overall response went on to undergo transplant procedures, and those receiving revumenib maintenance post-transplant remained in remission at the time of data cutoff. The study noted no treatment-related discontinuations or dose reductions, underscoring revumenib’s tolerability and potential as an effective treatment option for this high-risk patient group. These findings offer new hope for pediatric patients who have exhausted other therapies, highlighting the promise of revumenib as a novel therapeutic approach in challenging cases of KMT2Ar acute leukemia.
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