Pancreatic cancer, one of the most difficult to treat and deadliest malignancies, now has a new FDA-approved therapy that helps patients live longer, a first-in-class Revolution Medicines drug that blocks mutated proteins driving this disease.
The once-daily pill, known in development as daraxonrasib, will reach the market under the brand name Rasonque. The FDA’s Wednesday regulatory decision covers the treatment of adults who have received at least one prior systemic therapy for the most common type of pancreatic cancer, where the standard of care is chemotherapy. Such patients must also not be candidates for a multi-agent systemic therapy.
Rasonque is a small molecule that targets RAS, a protein that acts like an on/off switch to regulate processes such as cell growth and differentiation. Mutations can result in RAS proteins that are stuck in the “on” position, contributing to the unregulated cell growth that drives cancer.
While scientists have long known the role that mutated RAS plays in various cancers, the protein has been an elusive one to drug. The first approved RAS inhibitors, molecules marketed by Amgen and Bristol Myers Squibb, only block a specific mutation called KRAS G12C. These drugs target the protein in its inactive or “off” state, preventing it from becoming active. Redwood City, California-based Revolution, or RevMed for short, took a different tack, designing its drug to block multiple forms of RAS that are in the “on” state.
RevMed’s most advanced program for Rasonque is pancreatic adenocarcinoma, which is cancer that starts as tumors in the cells lining the ducts of the pancreas. About 67,500 new cases of pancreatic cancer are diagnosed in the U.S. annually, according to the National Cancer Institute. Of these cases, an estimated 90% to 95% are pancreatic adenocarcinoma, making it the most common type of pancreatic cancer. In addition to being a hard cancer to detect, it’s also aggressive. Many cases aren’t diagnosed until the disease has advanced, making it difficult to treat and particularly deadly.
Rasonque’s regulatory submission was based on an open-label Phase 3 trial that enrolled 500 adults with previously treated metastatic pancreatic adenocarcinoma. The main goals were measuring overall survival and progression-free survival. In April, RevMed reported preliminary results showing a median 13.2 months overall survival for the study drug arm compared to a median 6.7 months in the group that received standard of care chemotherapy, a near doubling of survival in a disease where the second-line treatment has been more chemo.
More details came during the annual meeting of the American Society of Clinical Oncology (ASCO) in May. Median progression-free survival was 7.2 months in the study drug arm compared to 3.6 months for patients who received standard chemo. When the trial’s lead investigator detailed the data during the ASCO presentation, attendees responded with a rare standing ovation. The most common side effects of Rasonque include rash, diarrhea, inflammation of the mucus membranes of the mouth, nausea, fatigue, and vomiting. Concurrent with the ASCO presentation, detailed results were published in the New England Journal of Medicine.
The FDA accepted RevMed’s Rasonque new drug application late last month. Applications typically receive a regulatory decision in 10 months. Priority review can cut that down to six months. Rasonque’s review was accelerated with a Commissioner’s National Priority Voucher, a pilot program that shaves the review time to one or two months for medicines in the national interest, such as difficult-to-treat cancers with limited therapeutic options. RevMed’s drug, formerly known by the code name RCM-6236, received a voucher last fall.
“Today’s approval provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer,” acting FDA Commissioner Kyle Diamantas said in the agency’s announcement of the regulatory decision. “It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible.”
RevMed set a $39,800 list price for a 30-day supply of Rasonque, which the company said is now available. In its report of second quarter 2026 financial results earlier this month, the company said it has been preparing for a commercial launch of the drug in the U.S. and around the world. The European Medicines Agency is currently evaluating this drug under a process that could expedite a regulatory decision. As of June 30, RevMed reported its cash position was $3.9 billion.
Clinical trials are ongoing that could expand the use of daraxonrasib. In pancreatic cancer, Phase 3 trials are ongoing evaluating the drug as a first-line treatment and as an adjuvant, a therapy that prevents cancer recurrence after it has been surgically removed. A pivotal study is ongoing testing the molecule as a second- or third-line treatment for metastatic non-small cell lung cancer. Phase 1/2 studies are ongoing testing the drug in combination with other therapies as a treatment for various types of solid tumors.
Photo by Revolution Medicines
