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The Food and Drug Administration (FDA) has approved Rasonque (daraxonrasib), a new daily pill that nearly doubles survival time for adults with advanced pancreatic cancer who have exhausted standard treatments. According to the FDA's announcement, the approval marks a major breakthrough for a disease that is among the deadliest cancers in the United States, often diagnosed late and with limited treatment options.
Rasonque is the first drug approved to target multiple forms of a protein called RAS, a main driver of tumor growth in pancreatic adenocarcinoma, which accounts for up to 95 percent of the approximately 67,000 pancreatic cancer cases diagnosed each year in the U.S. The FDA granted the drug both breakthrough therapy and orphan drug designations, expediting its review under the Commissioner’s National Priority Voucher pilot program. Approval came about six and a half months ahead of the standard review deadline, reflecting the urgency and impact of the therapy. The FDA’s official release highlights these regulatory milestones.
A late-stage clinical trial involving 500 patients showed that those receiving Rasonque lived a median of 13.2 months, compared to 6.7 months for those treated with standard chemotherapy. The study also found improved progression-free survival for patients on the new pill. As reported by The New York Times, some patients on Rasonque have seen their tumors shrink dramatically, and several have lived for years after diagnosis. Dr. Anna Berkenblit, chief scientific and medical officer at the Pancreatic Cancer Action Network, said, "We've never seen a benefit like this."
Since May, more than 2,000 patients received early access to Rasonque through an expanded access program. Revolution Medicines stated that insurance coverage is expected for most patients, although the final out-of-pocket costs are not yet clear. Wall Street analysts predict the annual price will be several hundred thousand dollars, but most of the cost will be covered by insurance plans.
Rasonque’s approval follows decades of research into targeting KRAS mutations, long considered an “undruggable” protein. The drug works by binding to and disabling the mutated RAS protein, cutting off a critical source of tumor growth. Researchers say this approach may open the door to similar treatments for other cancers driven by KRAS mutations, including certain lung and colon cancers. Becker’s Hospital Review reports that a phase 3 trial is underway to test the drug for use earlier in the course of pancreatic cancer treatment.
While Rasonque is not a cure, oncologists and patient groups have called it the most significant advance for pancreatic cancer in decades. Side effects can include rash, diarrhea, fatigue, and nausea, and some patients in trials reported harsh side effects. However, the extension in survival has brought hope to patients and families facing a disease where five-year survival rates remain below three percent for those with metastatic cancer.
Looking ahead, Revolution Medicines and other companies are developing additional RAS-targeting drugs and testing them in new trials. If ongoing studies confirm current results, Rasonque could become available to more patients and be used as a first-line treatment.