US approves pancreatic cancer pill that nearly doubled median survival in trial

A once-daily pill has become the first approved therapy in the United States to target multiple forms of RAS proteins, a long-frustrating driver of pancreatic cancer. The Food and Drug Administration approved daraxonrasib, sold as Rasonque, for a defined group of adults with metastatic pancreatic adenocarcinoma who have received at least one previous drug treatment or cannot undergo multidrug therapy.

The decision followed a randomized, multicenter trial involving 500 previously treated patients across North America, Europe and Asia. Median overall survival reached 13.2 months among those taking daraxonrasib, compared with about 6.7 months for those receiving chemotherapy. Median time without disease progression increased from 3.6 to 7.2 months. Tumors clearly shrank or disappeared in 31.6 percent of patients taking the pill, versus 11.2 percent in the chemotherapy group.

Daraxonrasib blocks several forms of RAS proteins, whose mutations can keep growth signals switched on inside cancer cells. Mutations in the related KRAS gene occur in more than 90 percent of pancreatic cancers but have proved difficult to target for decades.

The treatment is not a cure and is not approved for prevention or early-stage disease. Common side effects include rash, diarrhea, nausea, fatigue and vomiting, while bleeding is another potential risk. Even with those limitations, the approval adds an important option for a particularly hard-to-treat group.