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) of revumenib. This drug is intended for the treatment of adult and pediatric patients with relapsed or refractory (R/R) KMT2A-rearranged acute leukemia. The new action date is set for December 26, 2024.
The FDA stated on July 26, 2024, that the extension was necessary to allow sufficient time to fully review supplemental information provided by Syndax in response to the agency's requests. The submission of this additional data was classified as a Major Amendment to the NDA, resulting in the standard three-month extension. Syndax has confirmed that the FDA has not requested any additional trials or manufacturing information.
Significance of Revumenib
According to Michael A. Metzger, Chief Executive Officer of Syndax, "Revumenib, upon approval, will be the first drug indicated to treat patients with KMT2A-rearranged acute leukemia, a population with significant unmet need." The company remains confident that the data from the AUGMENT-101 trial, along with the additional information submitted, will support approval and demonstrate the significant benefits of revumenib for patients with this aggressive form of leukemia.
The NDA for revumenib has been granted Priority Review and is being reviewed under the FDA's Real-Time Oncology Review (RTOR) program. The FDA had previously granted Breakthrough Therapy, Fast Track, and Orphan Drug designations for revumenib.
About Revumenib
Revumenib is a selective, small molecule inhibitor targeting the menin-KMT2A binding interaction. It is under development for the treatment of KMT2A-rearranged (KMT2Ar) acute leukemias, also known as mixed lineage leukemia rearranged or MLLr, including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), as well as mutant nucleophosmin (mNPM1) AML. Topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia, which met its primary endpoint, were presented at the 65th American Society of Hematology Annual Meeting. Data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib has received Orphan Drug Designation for AML and ALL from the FDA and for AML from the European Commission, as well as Fast Track designation from the FDA for R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. The FDA also granted Breakthrough Therapy Designation for R/R acute leukemia with a KMT2A rearrangement.
Real-Time Oncology Review Program
The Real-Time Oncology Review (RTOR) program aims to expedite the review process for oncology drugs, ensuring timely access to safe and effective treatments for patients. This program facilitates close engagement between the sponsor and the FDA throughout the submission process, allowing the FDA to review individual sections of a drug application rather than requiring complete modules upfront.
