The Food and Drug Administration announced Wednesday that it has granted expedited approval to daraxonrasib, a novel oral therapy for the most common form of pancreatic cancer. The drug, marketed by Revolution Medicines under the brand name Rasonque, targets KRAS mutations that drive tumor growth in more than 90% of pancreatic cancer cases.
How the drug works
Daraxonrasib acts as a molecular “glue,” binding to multiple KRAS subtypes and preventing the mutated protein from signaling cancer cells to proliferate. This approach has long been considered “undruggable,” making the approval a landmark achievement in oncology research.
Clinical trial results
In a company‑funded, randomized study of 500 patients with metastatic pancreatic cancer that had stopped responding to prior therapy, those receiving daraxonrasib lived a median of 13.2 months, compared with 6.7 months for patients treated with standard chemotherapy. The trial also reported fewer severe adverse events; the most common side effects were skin rash, mouth sores, diarrhea and other digestive issues.
“This is one of the most anticipated approvals I can think of,” said Dr. Andrew Coveler of the Fred Hutch Cancer Center. “The medicine is not a cure but a significant improvement.”
Cost and access
Revolution Medicines said a one‑month supply of Rasonque will cost approximately $39,800. Earlier this year, former Senator Ben Sasse (R‑Neb.) shared his personal experience on CBS’s “60 Minutes,” noting reduced pain while taking the experimental drug. Public interest prompted the FDA to allow expanded access for patients meeting specific criteria before the formal approval.
Impact on the broader fight against cancer
Doctors and researchers view the approval as a potential gateway to additional therapies targeting KRAS mutations in other tumor types. Dr. Pashtoon Kasi of City of Hope Orange County remarked, “I think this has opened doors for many other companies. Downstream we will see more trials exploring this approach in other cancers.”
Revolution Medicines, based in Redwood City, California, is already investigating the molecular‑glue technology for lung cancer and other malignancies, suggesting that the breakthrough could have far‑reaching implications beyond pancreatic disease.
National context
Pancreatic cancer remains one of the deadliest cancers in the United States, with the American Cancer Society estimating about 67,000 new diagnoses and more than 52,000 deaths this year. The five‑year overall survival rate sits at roughly 13%, underscoring the urgent need for more effective treatments.
FDA Acting Commissioner Kyle Diamantas emphasized the agency’s commitment to rapid delivery of life‑saving therapies, noting that the approval came more than six months ahead of the agency’s original target date.
While the high price of Rasonque raises questions about affordability, the drug’s demonstrated survival benefit and tolerability represent a meaningful step forward for patients and families confronting a devastating diagnosis.
Original reporting: NBC Connecticut — read the source article.
