Syndax Extends PDUFA Date for Revumenib NDA in Relapsed/Refractory KMT2Ar Acute Leukemia

Syndax Pharmaceuticals, a clinical-stage biopharmaceutical company focused on developing innovative cancer treatments, has 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) of revumenib. This drug is intended for adults and pediatric patients with relapsed or refractory (R/R) KMT2Ar acute leukemia. The new PDUFA action date is set for December 26, 2024.

The FDA communicated to Syndax on July 26, 2024, that they required additional time to thoroughly review supplementary information provided by the company. This additional information submission led to the classification of a Major Amendment to the NDA, extending the initial PDUFA action date from September 26, 2024, by three months. Importantly, the FDA has not requested any additional clinical trials or manufacturing data.

Michael A. Metzger, Chief Executive Officer of Syndax Pharmaceuticals, expressed confidence in revumenib's potential to meet a significant unmet medical need. He highlighted the strength of the data from the AUGMENT-101 trial and the additional information provided to the FDA, emphasizing the potential of revumenib to bring meaningful benefits to patients suffering from this severe form of leukemia. Metzger stated that the company looks forward to continued engagement with the FDA as they finalize their review by the newly set date in December.

The NDA for revumenib was granted Priority Review and is being evaluated under the FDA's Real-Time Oncology Review (RTOR) program. Previously, the FDA granted Breakthrough Therapy, Fast Track, and Orphan Drug designations for revumenib, underlining the importance and potential impact of this drug.

Revumenib is a potent, selective small molecule inhibitor targeting the menin-KMT2A binding interaction. It is being developed for treating various acute leukemias, including KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged (MLLr), acute leukemias, and mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). The Phase 2 AUGMENT-101 trial showed positive topline results, meeting the primary endpoint for patients with R/R KMT2Ar acute leukemia. These results were presented at the 65th American Society of Hematology Annual Meeting, with Phase 1 data also published in the journal Nature.

Revumenib has received Orphan Drug Designation for treating AML and ALL from both the FDA and the European Commission. Additionally, the FDA granted Fast Track designation for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. The FDA also awarded Breakthrough Therapy Designation for revumenib for treating adult and pediatric patients with R/R acute leukemia featuring a KMT2A rearrangement.

The Real-Time Oncology Review (RTOR) program aims to streamline the review process for oncology drugs, ensuring that safe and effective treatments reach patients as swiftly as possible. This program involves early and continuous engagement between the sponsor and the FDA, allowing the review of individual sections of the application as they are submitted, rather than waiting for the completion of the entire application.

Syndax Pharmaceuticals continues to advance its innovative pipeline of cancer therapies, with revumenib being a key focus. The company remains dedicated to addressing significant unmet medical needs in oncology and improving outcomes for patients battling devastating diseases like acute leukemia.