FDA approves once-daily pill that doubles survival in advanced pancreatic cancer

The U.S. Food and Drug Administration has approved daraxonrasib, a once-daily oral medication, as a new treatment for the most common form of pancreatic cancer. The approval, announced on August 26, follows clinical trial results that showed the drug doubled survival in patients with stage four disease who had already received chemotherapy.

Dr. Rachna Shroff, chief of hematology oncology at the University of Arizona Cancer Center, called the drug a "holy grail" in cancer research. Pancreatic cancer is often diagnosed late because its symptoms — pain, weight loss, reduced appetite, and jaundice — are nonspecific. More than 80 percent of cases are found at advanced stages.

Daraxonrasib targets the RAS signaling pathway, which is mutated in over 90 percent of pancreatic cancer cases. Unlike earlier attempts, this drug works regardless of whether a RAS mutation is present. It acts as a molecular glue, attaching to the RAS protein and cutting off the signal that drives uncontrolled cancer growth.

In a large phase-three trial, patients taking daraxonrasib lived a median of 13.2 months, roughly double the survival seen with other chemotherapies. The drug is the first chemotherapy-free option for these patients, taken as a single pill daily rather than through IV infusions.

Side effects include rash and diarrhea, but doctors have learned to manage them with preventive medications and dose adjustments. Patient-reported outcomes showed improved quality of life compared with chemotherapy, with fewer patients discontinuing treatment.

RAS alterations occur early in pancreatic cancer development, making the pathway a key target. The drug's success has spurred research into other cancers. Early September data showed promising results in non-small cell lung cancer, and trials are ongoing across multiple tumor types. Researchers describe this as the beginning of a broader shift toward targeting RAS in oncology.