The U.S. Food and Drug Administration on Wednesday approved Rasonque (daraxonrasib), a first-in-class targeted therapy for adults with metastatic pancreatic cancer who have received prior treatment or cannot tolerate combination chemotherapy. The approval marks a significant advance in a disease with a five-year survival rate of approximately 13%, one of the lowest among major cancers.
The drug, developed by Revolution Medicines, is a once-daily oral pill designed to block multiple forms of the RAS protein, which drives tumor growth in many pancreatic cancers. The FDA granted the approval under its National Priority Voucher program, accelerating the review process from the typical 10–12 months to just one to two months.
Key trial results
The approval was based on a phase 3 randomized clinical trial involving 500 adults with previously treated metastatic pancreatic adenocarcinoma, the most common type of pancreatic cancer. Patients receiving Rasonque experienced a median overall survival of 13.2 months, compared to 6.7 months for those receiving standard chemotherapy. The drug also demonstrated a more favorable side effect profile, with common adverse reactions including rash, diarrhea, mouth inflammation, nausea, fatigue, vomiting, abdominal pain, swelling, reduced appetite, and bleeding.
Regulatory pathway and access
The FDA previously granted early access to Rasonque under its Expanded Access Program, allowing patients with serious or life-threatening conditions to receive the experimental treatment prior to formal approval. Revolution Medicines has not yet announced pricing or availability details.
Disease background
Pancreatic cancer accounts for about 3% of all cancer cases and 8% of all cancer deaths in the U.S., according to the American Cancer Society. Survival rates vary significantly by stage: approximately 44% for localized tumors, but dropping to 3% for metastatic disease. The cancer is often diagnosed at advanced stages due to a lack of early symptoms.
Clinical significance
The approval represents a breakthrough in targeting the RAS protein, a mutation present in over 90% of pancreatic cancers that had long eluded pharmaceutical research. The drug’s mechanism offers a new therapeutic approach for a historically difficult-to-treat malignancy. Former Sen. Ben Sasse (R-Neb.), a trial participant, has publicly credited the drug with reducing his tumor burden after a terminal diagnosis.