The U.S. Food and Drug Administration (FDA) has granted accelerated approval to a groundbreaking cancer drug. The experimental pill, daraxonrasib, developed by the biotechnology company Revolution Medicines, showed remarkable results in early clinical trials. In the first trial involving 83 patients with metastatic pancreatic cancer, tumors shrank in 29% of patients, while 90% saw no new tumor growth.
The average survival time increased to 15.6 months, roughly double the expected lifespan for this condition. In one patient, tumors shrank by 64% after starting treatment. Debbie Orcutt, a 69-year-old patient, told The Washington Post, "I had nothing to lose. I decided to try the trial."
In October, the FDA granted Revolution Medicines' drug an accelerated review under the "Commissioner's National Priority Voucher," which can reduce review time from a year to just one month. Other drugs targeting fertility, type 1 diabetes, hearing loss, blindness, addiction, and rare blood disorders have also received priority review under this program.
Andrew Nixon, a spokesperson for the U.S. Department of Health, said the process would be "accelerated without compromising scientific rigor."
Daraxonrasib targets mutations in the RAS gene family, which act as "on-off" switches controlling cell growth. Mutated RAS genes can cause uncontrolled cell proliferation. In 2013, Revolution Medicines co-founder Prof. Kevan Shokat developed a mechanism to stop the signaling disruptions caused by these genes and later adapted it to other proteins. CEO Mark Goldsmith said, "Many scientists thought this would destroy healthy cells, but animal studies showed no toxic effects."
While daraxonrasib has extended survival for some patients, it is not yet considered a definitive treatment. In another study with 40 patients, 95% experienced side effects and 35% had severe side effects. Dr. Brian Wolpin of Dana-Farber Cancer Institute said, "This drug could be a milestone in pancreatic cancer research," noting it shrinks tumors and eases patient pain.
Pancreatic cancer is the third leading cause of cancer deaths in the U.S., with only 13% of patients surviving more than five years. For metastatic cases, survival drops to just 3%. Anna Berkenblit, head of the Pancreatic Cancer Action Network, welcomed the accelerated approval, saying, "Targeting RAS mutations with this drug is a real leap forward in pancreatic cancer treatment."
Pranati Perati, a 55-year-old molecular biologist who began treatment in 2023 after other therapies failed, said the drug stopped tumor growth for two years, giving her "extra time to live." The FDA's accelerated approval system is expected to allow earlier access to new cancer drugs, though experts caution that speed should not compromise scientific validation.
