FDA Approves Targeted Pill for Metastatic Pancreatic Cancer
The US Food and Drug Administration (FDA) on Wednesday approved Revolution Medicines' daraxonrasib, marketed as Rasonque, for adults with metastatic pancreatic cancer who have received prior treatment or cannot receive combination chemotherapy. The once-daily pill, described as first-in-class, is designed to block several forms of the RAS protein, which helps drive tumor growth in many pancreatic cancers, according to reports from The Guardian and the Jerusalem Post.
The approval was based on a company-funded study of 500 adults with previously treated metastatic pancreatic adenocarcinoma, the most common type of the disease. Patients receiving the drug lived for a median of 13.2 months, compared with 6.7 months for those on standard chemotherapy—nearly doubling overall survival. The drug also showed fewer severe side effects, the reports said.
Expedited Review and Early Access
The FDA had previously granted early access to daraxonrasib under a program allowing patients with serious or life-threatening conditions to receive experimental treatments outside clinical trials. According to the Jerusalem Post, the drug was included in the FDA Commissioner's National Priority Voucher program, which accelerates reviews from the usual 10 to 12 months to as little as one to two months for therapies addressing major US health priorities and substantial unmet medical needs. The Guardian reported that FDA regulators noted the approval came more than six months ahead of their target date.
"It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible," FDA acting commissioner Kyle Diamantas said in a statement, as quoted by The Guardian.
The drug garnered public attention earlier this year when former US senator Ben Sasse of Nebraska described on CBS's 60 Minutes how he has had less pain while taking it. The Guardian reported that a surge in public interest led the FDA to allow expanded access before official approval for patients meeting certain criteria.
Significance in a Deadly Disease
Pancreatic cancer is among the most deadly cancers, largely because it is hard to detect before spreading. The American Cancer Society estimates about 67,000 new cases will be diagnosed in the US this year and more than 52,000 people will die from the disease, according to The Guardian. The five-year survival rate is about 13%, one of the lowest among major cancers, both outlets noted.
Daraxonrasib blocks a mutated protein that fuels tumor growth in more than 90% of pancreatic cancer cases, an approach that had eluded drugmakers for decades, The Guardian reported. The drug uses what is essentially a molecular glue to bind with multiple subtypes of the Kras mutation, which is especially critical in fueling pancreatic cancer. Revolution Medicines, based in Redwood City, California, is also studying the technology in other cancers, including lung cancer, according to that report.
Side Effects and Next Steps
Common side effects associated with daraxonrasib included rash, diarrhea, mouth inflammation, nausea, tiredness, vomiting, abdominal pain, swelling, reduced appetite, and bleeding, both outlets reported. Revolution Medicines did not immediately respond to a Reuters request for comment on pricing and availability, the Jerusalem Post noted.
Doctors who treat pancreatic cancer have expressed optimism that the approval may usher in more new options, with dozens of experimental drugs in development, The Guardian reported—though no additional details were provided in the coverage seen.