SILVER SPRING, Md. — The Food and Drug Administration on Wednesday approved daraxonrasib, a daily pill from Revolution Medicines that in late-stage trials nearly doubled median survival for patients with advanced pancreatic cancer, one of the deadliest and historically hardest-to-treat malignancies.
Sold under the brand name Rasonque, the drug will list at about $39,800 for a one-month supply, the company said in an announcement covered by CBS News. The FDA cleared it more than six months ahead of the target review date after granting it Breakthrough Therapy designation in 2025.
The trial
In a company-funded, randomized study of 500 patients whose metastatic disease had stopped responding to prior treatment, the pill produced a median overall survival of 13.2 months versus 6.7 months for those on additional chemotherapy, according to CBS. The BBC, citing the same trial, reported the chemotherapy median at 6.6 months; both accounts described the near-doubling as unusual for the disease.
Severe side effects were somewhat less common than with chemotherapy: 44 percent of daraxonrasib patients experienced them, compared with 57.5 percent of chemotherapy patients, according to the BBC. Common effects included rash, diarrhea, nausea, fatigue and vomiting, the BBC said; CBS added mouth sores.
The KRAS problem
Daraxonrasib blocks a mutated protein made by the KRAS gene, which fuels tumor growth in more than 90 percent of pancreatic cancers, both outlets reported.
"Most patients get the diagnosis of pancreatic cancer quite late, at which point its already metastatic, it's already spread," Dr. Céline Gounder, CBS News's medical correspondent, said on-air. "… This is a pill that blocks a protein called RAS, and mutations in RAS drive around 90% of all pancreatic cancers. Scientists have been calling RAS 'undruggable' for the last 40 years, and now finally, we have a drug for it."
Angelo de Claro, director of the FDA's Oncology Center of Excellence, said the agency granted expedited approval because the drug "showed unprecedented results in an area of high unmet need," according to the BBC. It provides, the FDA said, "a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer."
The scale of the disease
The American Cancer Society estimates about 67,000 new pancreatic cancer cases will be diagnosed in the United States this year, and more than 52,000 people will die from the disease, CBS reported. The five-year survival rate is 13 percent. More than half of pancreatic cancer patients die within three months of diagnosis, per the BBC.
Former Sen. Ben Sasse, who disclosed in December that he had been diagnosed with Stage 4 pancreatic cancer, is a trial participant. On "60 Minutes" in April, Sasse described the pill as a "miracle drug" and said he had seen a "massive 76% reduction in tumor volume over the last four months," according to CBS's account. Sasse, given a three-to-four-month life expectancy at diagnosis, said, "I am on extended time already."
Caveats
The drug is not a cure. "This medicine is not a cure but a significant improvement," Dr. Andrew Coveler of the Fred Hutch Cancer Center told CBS, calling the approval one of "the most anticipated" he could recall. The $39,800 monthly list price will test what U.S. commercial insurers and Medicare are willing to cover, and access outside the United States is not addressed by Wednesday's decision.
"It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible," Kyle Diamantas, the FDA's acting commissioner, said in a statement quoted by CBS.
Revolution Medicines said it would begin distribution immediately. Its next trials will test the pill earlier in the disease course and in combination with other agents.