GlobeNewswire·Oct 30, 2026, 9:01 AM ET

Syndax’s Revuforj® (revumenib) Wins Prestigious 2026 Prix Galien USA Award for Best Product for Rare/Orphan Diseases

SNDX

Revuforj (revumenib) was named Best Product for Rare/Orphan Diseases of 2026 by The Galien Foundation.
Revuforj is the first & only FDA-approved menin inhibitor for adults & children aged one year and older with relapsed or refractory (R/R) NPM1-mutated AML or R/R KMT2A-translocated acute leukemia.
FDA approval for R/R KMT2A-translocated leukemia was granted in November 2024 & expanded to include NPM1-mutated AML patients in October 2025.
Differentiation syndrome is a fatal risk associated with Revuforj, occurring in 25% of patients in clinical trials.
QTc interval prolongation & Torsades de Pointes are significant risks, reported in 36% of patients treated with Revuforj.
Fatal adverse reactions occurred in 4% of patients, including sudden death, differentiation syndrome, hemorrhage, & cardiac arrest.
Common adverse reactions include increased phosphate (51%), hemorrhage (48%), nausea (48%), & various infections.
Revuforj requires dose adjustments or discontinuation when used with strong CYP3A4 inhibitors or QTc-prolonging drugs.
The drug poses risks of fetal harm & potential reversible infertility, necessitating contraception during treatment & for 4 months post-therapy.
Pediatric patients require monitoring for bone growth & development, while geriatric patients have higher incidences of QTc prolongation & edema.
Syndax Pharmaceuticals, the commercial-stage biopharmaceutical company behind Revuforj, continues to evaluate the drug in combination therapies for newly diagnosed patients.