FDA grants early access to experimental pancreatic cancer pill

The Food and Drug Administration has opened an early-access pathway for daraxonrasib, an experimental pancreatic cancer pill that could reach some patients before full approval if their doctors and the company agree it is appropriate. The move is aimed at people with previously treated metastatic pancreatic ductal adenocarcinoma, one of the hardest forms of the disease to treat, and it comes as patients and oncologists weigh urgent need against the limits of the available evidence.

The agency issued a “safe to proceed” letter to Revolution Medicines on April 30, after receiving the company’s expanded-access request two days earlier, on April 28. Expanded access is not the same as approval. It is the FDA route for patients who cannot enroll in a clinical trial and have no satisfactory therapeutic options, a system the agency says it has used for experimental drugs and biologics since the 1970s. Revolution Medicines said patients must work through their treating physician, and the company said it is moving as quickly as possible to make access safe and equitable for eligible patients in the United States.

Daraxonrasib, also known as RMC-6236, is an oral multiselective RAS(ON) inhibitor being studied across several RAS-driven cancers, including pancreatic, lung and colorectal cancer. Its profile has drawn intense attention because more than 90% of pancreatic tumors carry a KRAS mutation, according to the National Cancer Institute, making the pathway a central target in the hunt for better treatments. The American Cancer Society puts the overall five-year relative survival rate for pancreatic cancer at 13%, a stark measure of how little room remains for delay in this disease.

The timing of the FDA action matters because Revolution Medicines reported positive topline results from its phase 3 RASolute 302 trial on April 13, saying the study met its primary and key secondary endpoints, including progression-free survival and overall survival. One summary of the data said median overall survival was 13.2 months with daraxonrasib versus 6.7 months with chemotherapy in previously treated metastatic PDAC. Those findings, while encouraging, will still face scrutiny when detailed results are presented at ASCO’s annual meeting in Chicago from May 29 to June 2.

For patients and advocates, the expanded-access decision is already landing as a sign of momentum in a field that has long offered too few options. Former Sen. Ben Sasse, who has advanced pancreatic cancer, called daraxonrasib “a miracle drug” and said it was helping him live longer and with less pain. For regulators, the step underscores a familiar test of cancer policy: how to respond when early data look promising, the need is immediate, and the final balance of benefit and risk is still being assembled.