FDA approves first KRAS-targeted pill for pancreatic cancer

Daraxonrasib approval came more than six months ahead of FDA’s target date

The U.S. Food and Drug Administration on Wednesday granted expedited approval to daraxonrasib, the first treatment to directly target the Ras gene mutations that drive most pancreatic cancers, the agency said. The once-daily pill, developed by Revolution Medicines and to be sold under the brand name Rasonque, is the first therapy to overcome a structural obstacle that has kept the cancer-driving protein considered “undruggable” for decades.

In a company-funded trial that randomly assigned 500 patients with metastatic pancreatic cancer that had stopped responding to prior treatment, those who received daraxonrasib lived a median of 13.2 months, compared with 6.7 months for those who received additional chemotherapy. Patients on the new drug also experienced fewer severe side effects, according to the study.

The approval covers the most common form of pancreatic cancer, in which mutated KRAS proteins fuel tumor growth in more than 90% of cases. Revolution Medicines’ drug uses what the company describes as a molecular glue to bind with multiple KRAS subtypes simultaneously — an approach that has eluded drugmakers since the mutation’s role in cancer was first understood.

“It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible,” the FDA’s acting commissioner, Kyle Diamantas, said in a statement. The agency approved the drug more than six months ahead of its target date.

Pancreatic cancer remains among the deadliest common cancers in part because it is difficult to detect before it spreads to other organs. The American Cancer Society estimates that roughly 67,000 new cases will be diagnosed in the United States this year and more than 52,000 people will die from the disease. The five-year overall survival rate stands at 13%.

The drug drew broader public attention earlier this year when former U.S. Senator Ben Sasse of Nebraska described on CBS’s “60 Minutes” program how he had experienced less pain while taking it. The surge in interest led the FDA to allow expanded access — making the drug available before official approval to patients who met certain criteria.

Doctors who treat pancreatic cancer have expressed optimism that the approval could open the door to additional treatment options. Unlike with other cancers that have benefited from multiple chemotherapy alternatives, pancreatic cancer has been harder to tackle, and dozens of experimental drugs are now in development.

Revolution Medicines, based in Redwood City, California, is studying the same molecular-glue technology against other cancers, including lung cancer, where KRAS mutations also play a significant role.