In April, the FDA granted early access to daraxonrasib, an experimental drug so promising that some physicians are already calling it a miracle—and pancreatic cancer clinics across the United States are now overwhelmed with patient requests they can barely handle.
Daraxonrasib, made by Revolution Medicines, targets the genetic root of the disease itself. Over 90% of pancreatic cancer patients carry a mutation in the KRAS gene, and this once-daily tablet works by binding to and silencing that faulty genetic switch. As Dr. Christopher Lieu of the University of Colorado Anschutz Medical School explained the mechanism, "The drug binds to the activating pocket of the gene mutation and shuts it down. It's almost like if you have a bullhorn and you cover it up so no sound can escape."
The numbers tell why the excitement is warranted. In late clinical trials, patients taking daraxonrasib lived a median of 13.2 months—nearly double the 6.7 months achieved with standard chemotherapy. This matters urgently because pancreatic cancer is among the deadliest cancers known. Only 3% of patients with metastatic pancreatic cancer survive five years after diagnosis, according to the National Cancer Institute. This year alone, around 67,000 Americans will be diagnosed with pancreatic cancer, and roughly 53,000 will die from it, according to the American Cancer Society.
The drug's approval for early access on April 30 opened a door that patients have rushed through. Dr. Daniel King, a medical oncologist at Northwell Health's Zuckerberg Cancer Center, told Reuters that "the public caught wind of the FDA announcement, which has triggered a deluge of patient requests." Cancer centers across the country are now scrambling to open up protocols and provide access, he said. The expanded access program specifically targets patients whose pancreatic cancer has metastasized after previous treatment—people, in other words, with few other options.
The process to obtain the drug requires a licensed physician to submit a request to Revolution Medicines, where an institutional review board reviews it. The drugmaker expects to respond within two business days. If approved, the case goes to the FDA, and patients enter hospital monitoring to track side effects and outcomes. The drug is being offered for free during this phase.
What sets daraxonrasib apart is not just efficacy but the promise of fewer side effects. Because it targets the specific gene mutation driving the cancer rather than broadly poisoning cells, it may work with "potentially less toxicity" than chemotherapy, Dr. Lieu noted. That targeted approach—attacking the mechanism itself rather than the whole system—represents a fundamental shift in how pancreatic cancer might be treated.
Full FDA approval could come remarkably quickly. Under the FDA's expedited review process, daraxonrasib could receive full approval within one or two months of a formal application—far faster than the typical 10 to 12 months. Revolution Medicines has not yet filed that application but said in early May there is a "full-throttle effort" underway to do so. While the expanded access program was championed by former FDA Commissioner Dr. Marty Makary, who resigned in May, the agency has communicated no intended changes to the program.
For patients and families facing one of cancer's harshest diagnoses, that timeline matters. The deluge of requests at clinics reflects something deeper than clinical optimism: it reflects the arrival of genuine hope.