FDA Approves First Once-Daily Pill for Advanced Pancreatic Cancer With Doubled Survival Rates

Federal regulators granted approval Wednesday to Rasonque, a new oral medication designed to treat advanced pancreatic cancer. The drug, developed by Revolution Medicines based in California, works by inhibiting RAS proteins that fuel tumor development. The FDA expedited its review process, approving the treatment more than six months before its scheduled deadline.

Acting FDA Commissioner Kyle Diamantas emphasized the significance of the authorization in a statement. “Today’s approval provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer,” Diamantas said, adding that delivering cures to patients quickly remains the agency’s priority. He credited FDA scientists for conducting a rigorous yet accelerated review of the medication.

Rasonque targets adults with metastatic pancreatic cancer whose disease progressed despite prior treatment or who could not tolerate standard combination chemotherapy regimens. Pancreatic adenocarcinoma accounts for approximately 95 percent of roughly 67,000 annual pancreatic cancer diagnoses in the United States, according to the National Cancer Institute, making it one of the nation’s deadliest malignancies despite representing only 3.2 percent of all cancer cases.

A randomized clinical trial involving 500 patients demonstrated Rasonque’s efficacy compared to conventional chemotherapy. Patients receiving the once-daily pill achieved a median overall survival of 13.2 months, nearly doubling the 6.7-month median survival observed in the control group receiving standard chemotherapy. Angelo de Claro, director of the FDA’s Oncology Center of Excellence, called the results “unprecedented” for this area of significant medical need.

The agency granted Rasonque multiple designations to accelerate its development, including priority review status, orphan drug classification, and breakthrough therapy designation. The application also received evaluation through the FDA commissioner’s National Priority Voucher pilot program designed to expedite access to critical treatments. In May, regulators issued a “safe to proceed” letter enabling expanded access protocols that allowed patients to receive the medication before formal approval.

Common side effects documented during trials included rash, diarrhea, mouth sores, nausea, fatigue, vomiting, abdominal pain, fluid retention, reduced appetite, and bleeding. Anna Berkenblit, chief scientific and medical officer of the Pancreatic Cancer Action Network, hailed the approval as “the most significant advance we have seen in the fight against pancreatic cancer,” predicting the drug will improve patient outcomes and quality of life.