An experimental medication, daraxonrasib, has demonstrated significant clinical efficacy in treating advanced pancreatic cancer, effectively doubling patient survival time according to results from a Phase 3 trial. The findings were presented Sunday at the American Society of Clinical Oncology’s annual meeting in Chicago and concurrently published in the New England Journal of Medicine.
The study enrolled 500 participants who had previously exhausted standard chemotherapy options. Patients who received the drug lived an average of 13.2 months, while those who remained on chemotherapy lived an average of 6.7 months.
Dr. Zev Wainberg, co-director of the GI Oncology Program at UCLA Health and the trial's lead investigator, described the study as one of the most emotional of his career. Wainberg noted that every patient who entered his office expressed a preference for the experimental medication over enduring additional rounds of chemotherapy.
“Statistically, I knew only half of them get the pill, and we don’t get to choose,” Wainberg said. “I put a lot of patients on the chemo arm, and none of them are alive anymore.”
Dr. Rachna Shroff, chief of the division of hematology and oncology at the University of Arizona Cancer Center, characterized the results as a major breakthrough for the field. “It’s that big of a game-changer for those of us who treat pancreatic cancer,” Shroff stated. “It’s unprecedented.”
Because daraxonrasib targets a specific mutation in the KRAS gene, researchers are now investigating its potential utility for other malignancies. The drug, which is administered as three daily pills, is currently being evaluated for effectiveness against lung, colorectal, ovarian, and endometrial cancers, as well as cholangiocarcinoma, a form of bile duct cancer.
“Pancreas cancer may be the first for this drug, but there will be others,” said Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber Cancer Institute. “Now the floodgates open.”
The Food and Drug Administration has granted daraxonrasib fast-track status for pancreatic cancer. The agency has also authorized an expanded access program, allowing patients to receive the medication outside of the formal clinical trial environment.
Revolution Medicines, the manufacturer, has not yet announced a specific timeline for filing for full FDA approval. However, CEO Dr. Mark Goldsmith confirmed that company personnel are working to finalize the necessary data for submission.
