Syndax’s Revuforj Wins 2026 Prix Galien Rare Disease Award

New York, New York — October 30, 2026

Recognition of Targeted Treatment Innovation

The Prix Galien USA Award is presented by The Galien Foundation, which recognizes scientific and technological advances intended to improve human health. Syndax Chief Scientific Officer and Founder Peter Ordentlich, Ph.D., said the company was honored by the recognition and credited patients, researchers, clinicians and employees involved in bringing revumenib to patients. The award follows the medicine’s regulatory milestones in two genetically defined acute leukemia populations. NPM1 mutations are the most common genetic alteration in AML and occur in approximately 30% of adults with the disease. These mutations contribute to an aggressive cancer biology and are associated with substantial relapse risk. KMT2A rearrangements are another important genetic driver of acute leukemia and are associated with poor prognosis and high relapse rates. KMT2A translocations are estimated to occur in approximately 10% of acute leukemias and are particularly prevalent among infants and children with acute leukemia. The company describes Revuforj as the first and only approved menin inhibitor for the specified patient populations. Its ongoing and planned clinical development program includes evaluation in combination with standard-of-care therapies in newly diagnosed patients with NPM1-mutated or KMT2A-rearranged AML.

Clinical Safety Profile and Important Warnings

Revuforj carries a boxed warning for differentiation syndrome and QTc interval prolongation, including Torsades de Pointes. Differentiation syndrome can be life-threatening or fatal and may involve fever, breathing difficulties, low blood oxygen, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain, peripheral edema, low blood pressure and kidney dysfunction. In clinical trials involving 241 patients treated at the recommended dosage for relapsed or refractory acute leukemia, differentiation syndrome occurred in 25% of patients; 12% experienced Grade 3 or 4 events, and two cases were fatal. The median time to onset was nine days. The prescribing information directs clinicians to initiate systemic corticosteroids promptly when differentiation syndrome is suspected, alongside hemodynamic monitoring and supportive care. QTc prolongation was reported in 36% of patients, including Grade 3 events in 15% and Grade 4 events in 2%. One patient experienced cardiac arrest with a fatal outcome, and another experienced non-sustained Torsades de Pointes. Electrolyte abnormalities, including low potassium and magnesium, should be corrected before and during treatment. An ECG is required before treatment, at least weekly during the first four weeks and monthly thereafter. Revuforj should not be initiated in patients with a QTcF interval above 450 milliseconds. Other important precautions include embryo-fetal toxicity, potential fertility effects, and the need to monitor bone growth and development in pediatric patients. Common adverse reactions and laboratory abnormalities include increased phosphate, hemorrhage, nausea, infections, elevated liver enzymes, increased creatinine, musculoskeletal pain, febrile neutropenia and edema.

Expanded Development Across Acute Leukemia

Revuforj (revumenib) is approved for adults and pediatric patients aged one year and older with R/R AML carrying a susceptible NPM1 mutation who have no satisfactory alternative treatment options, or R/R acute leukemia with a KMT2A translocation, as determined by an FDA-authorized test. The medicine’s development program is continuing across different stages of the treatment landscape, including planned or ongoing combination studies with standard therapies in newly diagnosed NPM1-mutated and KMT2A-rearranged AML. Syndax also markets Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony-stimulating factor 1 receptor (CSF-1R). The company is pursuing additional clinical studies across its oncology pipeline. The Prix Galien recognition represents an industry award and does not change Revuforj’s approved indications, prescribing requirements or established safety precautions. Clinicians should consult the full prescribing information, including boxed warnings, for dosing, monitoring, contraindications and drug-interaction guidance. Important interactions include strong CYP3A4 inhibitors, strong or moderate CYP3A4 inducers and medicines that prolong the QTc interval. The award underscores the continued development of molecularly targeted therapies for genetically defined acute leukemia populations with significant unmet medical needs.

Source:Syndax Pharmaceuticals, Press release