FDA Expands Access to New Pancreatic Cancer Drug

Patients facing one of the deadliest cancers finally have a reason to hope, thanks to a drug that targets what scientists once thought was impossible to treat.

The FDA has authorized expanded access to daraxonrasib, an investigational drug that attacks the KRAS mutation driving over 90% of pancreatic cancer cases. For decades, researchers believed this genetic flaw was "undruggable" because it proved so difficult to stop with available medicines.

A major clinical trial published in The New England Journal of Medicine found that patients taking daraxonrasib went 8.5 months before their cancer worsened. That's significantly longer than what typically happens with standard chemotherapy for patients whose cancer has spread and stopped responding to earlier treatment.

Dr. Wungki Park, a gastrointestinal oncologist at Memorial Sloan Kettering Cancer Center who led the pancreatic cancer trial, says the results surprised even him. "Some patients reported that their symptoms improved just days after taking the drug," he explains. "We do not see that quick response from most chemotherapy regimens."

The trial included 168 patients at 16 cancer centers across the United States, with ages ranging from 30 to 86. All had stage 4 pancreatic cancer that had spread to other organs and had already gone through one round of chemotherapy that failed.

About 35% of patients responded to the drug, and overall survival reached 13.1 months. While side effects like rash, nausea, and mouth sores affected about one in three patients, no one stopped treatment because of them at the recommended dose.

Why This Inspires

Dr. Park describes treating a disease where time with loved ones is precious. His patients on daraxonrasib have attended daughters' weddings, celebrated grandchildren's births, and traveled again. "I feel like I am finally giving my patients the time they deserve with their loved ones," he says.

The drug works by essentially jamming the broken "go" signal that KRAS mutations create in cancer cells. It's effective against several types of KRAS mutations, meaning it could help a broader group of patients than previous targeted therapies.

After more than three decades of relying mainly on chemotherapy, pancreatic cancer treatment may finally be shifting. Patients who once faced limited options now have access to a drug that not only extends their lives but often improves how they feel while living them.