The Food and Drug Administration has approved a new drug called Rasonque to treat metastatic pancreatic adenocarcinoma, a form of cancer that has spread beyond the pancreas to other parts of the body. The approval marks a major shift in treatment options for a disease that has long resisted effective therapies.
According to a report by Healthline, pancreatic cancer was the 12th most common cancer in 2024, with more than 530,000 estimated diagnoses globally. It ranked as the sixth most common cause of cancer-related death, contributing to more than 490,000 deaths worldwide that year.
The FDA based its approval on results from a Phase 3 clinical trial. Patients receiving Rasonque had an average survival of 13.2 months, compared with 6.7 months for those who received chemotherapy alone. The drug is intended for patients who have already received systemic therapy, such as chemotherapy, and whose cancer has continued to progress.
Anton Bilchik, MD, PhD, a surgical oncologist and director of the Gastrointestinal and Hepatobiliary Program at Providence Saint John's Cancer Institute in Santa Monica, California, called the results historic. "This is probably the most exciting study that has been reported in pancreas cancer in the last 30, 40 years, if not forever," he said.
Bilchik described the results as striking given how difficult advanced pancreatic cancer is to treat. "It's truly astonishing that such patients with advanced pancreas cancer, who have a very poor outcome, can get a pill after they have failed first-line chemotherapy and have a doubling of survival, compared with those patients that just got standard chemotherapy," he told Healthline.
Rasonque works by targeting a family of genes called RAS. "RAS is a driver of pancreas cancer," Bilchik said. "RAS is present in more than 90% of the most common pancreas cancer, which is adenocarcinoma. RAS drives the change in cells from normal to abnormal, to becoming cancer."
Rosario Ligresti, MD, chief of gastroenterology at Hackensack University Medical Center in New Jersey, explained that these mutated RAS proteins have historically been considered difficult to treat because of their hard-to-bind surfaces. He described them as having been "undruggable" for that reason.
Ligresti said Rasonque gets around this problem through a novel mechanism. "Rasonque overcomes this by acting as a 'molecular glue' that first attaches to an abundant cellular chaperone protein called cyclophilin A," he told Healthline. That newly formed drug-chaperone complex then conforms to the active RAS protein, locking together to create what Ligresti called a "tri-complex" that physically blocks RAS from interacting with its downstream signaling partners. By targeting a broad spectrum of active RAS mutations rather than a single variant, the drug cuts off the signals that drive uncontrolled cancer cell growth.
The approval applies specifically to patients with metastatic pancreatic adenocarcinoma who have previously undergone systemic therapy. Rasonque is taken as a pill, which Bilchik noted is significant for patients managing advanced illness.
