Key Takeaways:
The Food and Drug Administration approved Revolution Medicines' Rasonque for advanced pancreatic cancer, a first-in-class therapy that nearly doubled survival to 13.2 months.
"This is life-altering," Carla Kurkjian, an oncologist at Mercy Hospital in Oklahoma City who wasn't involved in the studies, said. "Not just for patients, as far as helping them extend their lives beyond anything we'd ever seen, but for us as physicians, to be able to have a different conversation than we've had for so long."
The approval was supported by the Phase 3 RASolute 302 trial of 500 adults with previously treated metastatic pancreatic adenocarcinoma, the most common form of the disease. Patients who received Rasonque achieved a median overall survival of 13.2 months, compared with 6.7 months for those on standard chemotherapy. The once-daily pill blocks several forms of the RAS protein, a mutation that drives tumor growth in most pancreatic cancers.
Rasonque is Revolution Medicines' first approved drug. Evaluate projects it could generate more than $20 billion in annual sales if it proves safe and effective in earlier stages of the disease and other cancer types. The FDA granted the approval 6.5 months before the user fee deadline under the Commissioner's National Priority Voucher program.
RAS mutations drive as much as a third of all human cancers, including most pancreatic tumors. For four decades, researchers struggled to target the protein, whose flat surface lacks a pocket for conventional drugs to lock onto, leading some scientists to label it "undruggable." A breakthrough came in 2013 when Kevan Shokat, a chemical biologist at the University of California, San Francisco, found a druggable pocket on the protein. Shokat co-founded Revolution Medicines, which bought molecular-glue specialist Warp Drive Bio in 2018 and began testing the drug in patients in 2022.
The most common side effects included rash, diarrhea, mouth inflammation, nausea, tiredness, vomiting, abdominal pain, swelling, reduced appetite and bleeding. Former Nebraska Sen. Ben Sasse, who went public in the spring with what he described as a significant reduction in his tumors while in the trial, appeared on television with his face covered in red blotches.
The approval pressures competitors racing to develop their own RAS-targeting pancreatic-cancer drugs, including Eli Lilly and Pfizer. About 67,000 people in the U.S. are diagnosed with pancreatic cancer each year, and about seven in eight die within five years of diagnosis, according to the American Cancer Society.
Jay Williams, a 58-year-old former Disney executive, started taking Rasonque in July through an early-access pathway after chemotherapy, radiation and surgery failed to halt his cancer's spread. "What it does is buy you time, and that's one thing you don't always have with pancreatic cancer," he said.
The approval gives Revolution Medicines a foothold in a historically intractable disease and a pipeline anchor for expanding Rasonque into earlier treatment lines and other RAS-driven cancers. Investors will watch for the company's pricing announcement and enrollment in front-line trials that could unlock the $20 billion sales estimate.
This article is for informational purposes only and does not constitute investment advice.
