The U.S. Food and Drug Administration (FDA) has granted expedited approval to daraxonrasib, a new oral medication designed to treat advanced pancreatic cancer by targeting a specific protein mutation that drives tumor growth. This marks a significant development in treating one of the deadliest forms of cancer, offering a new therapeutic avenue for patients whose disease has progressed despite prior treatments.
Daraxonrasib, to be marketed as Rasonque by Revolution Medicines, targets mutated KRAS proteins, which are implicated in over 90% of pancreatic cancers. For decades, these mutations were considered “undruggable” due to the protein's structure. The drug functions as a molecular glue, binding to multiple KRAS subtypes. A one-month supply of the medication is priced at approximately $39,800.
In a company-funded study involving 500 patients with metastatic pancreatic cancer that had stopped responding to other treatments, those who received daraxonrasib experienced a median survival of 13.2 months. This nearly doubles the median survival of 6.7 months seen in patients who received standard chemotherapy. Side effects reported included skin rash, mouth sores, diarrhea, and other digestive issues, which were noted as fewer and less severe than those associated with chemotherapy.
Pancreatic cancer is particularly lethal due to its often late detection and propensity to spread. The American Cancer Society estimates approximately 67,000 new cases will be diagnosed in the U.S. this year, with over 52,000 deaths. The overall five-year survival rate currently stands at 13%.
Dr. Andrew Coveler of the Fred Hutch Cancer Center described the approval as highly anticipated, noting that while the drug is not a cure, it represents a substantial improvement in treatment options. The drug had previously gained public attention when former Sen. Ben Sasse discussed his experience with it on "60 Minutes," leading to expanded access for some patients prior to its official approval.
The FDA approved daraxonrasib more than six months ahead of its target date, a move acting commissioner Kyle Diamantas stated reflects the agency's commitment to delivering treatments to patients swiftly. The development of daraxonrasib may pave the way for new treatments for other cancers, as Revolution Medicines is investigating its technology for other cancer types, including lung cancer. Dr. Pashtoon Kasi of City of Hope Orange County expressed optimism that this approach will spur further research and clinical trials targeting similar mutations in other tumors.