The US Food and Drug Administration has approved Rasonque, a once-daily pill for advanced pancreatic cancer that roughly doubled survival in a late-stage trial. The drug, known chemically as daraxonrasib and made by Revolution Medicines, is the first targeted therapy of its kind to win approval for the disease.
A pill that targets a long-elusive protein
Pancreatic cancer tumors carry mutations in a gene called RAS in more than 90 percent of cases. For decades, drugmakers failed to block the mutated RAS protein, which fuels tumor growth. Daraxonrasib is a RAS inhibitor that finally attacks that target directly, a class of drugs once considered impossible to make.
The trial that won approval
In a randomized trial involving 500 adults with previously treated metastatic pancreatic adenocarcinoma, patients who took daraxonrasib lived a median of 13.2 months, compared with 6.7 months for those on standard chemotherapy. The approval covers patients who no longer respond to at least one earlier treatment or who cannot take multiagent chemotherapy.
Doctors described the result as a turning point for a disease with few options. Pancreatic cancer is the third leading cause of cancer death in the United States and the deadliest of all major cancers, with most patients diagnosed after the disease has spread.
What patients should know
Rasonque is a tablet taken once a day. The company says a one-month supply will cost about $39,800, and it plans to offer patient assistance programs. Researchers say the approval opens the door to combination treatments that pair RAS inhibitors with other therapies, and trials are already underway to test those combinations in earlier stages of the disease.
The approval also marks the first time a biomarker-driven therapy has shown an overall survival benefit in metastatic pancreatic cancer, according to the FDA. Researchers say the trial results support testing the drug in patients with earlier-stage disease, where treatment has a better chance of working. Several combination studies are already enrolling patients.