US cancer centers are grappling with an influx of patient requests for an experimental pancreatic cancer drug, daraxonrasib, as they navigate an early access program while awaiting what many hope will be swift Food and Drug Administration (FDA) approval.
FDA Greenlights Expanded Access Program
The FDA gave the green light to the expanded access program on 1 May, a mere three weeks after Revolution Medicines, the drug's developer, announced that its once-daily pill had doubled survival rates in a clinical trial for patients with advanced pancreatic cancer. This form of cancer is notoriously aggressive, with one of the lowest five-year survival rates. Revolution Medicines had sought permission from the FDA to provide the drug free-of-charge to patients with previously treated pancreatic cancer that had metastasized.
"The public caught wind of the FDA announcement... which has triggered a deluge of patient requests," said Dr. Daniel King, a medical oncologist at the Zuckerberg Cancer Center of Northwell Health. He added that cancer centers are now "all figuring out how to engage with our own institutions, opening up the protocols to provide access." Oncologists warn that establishing and managing such a program demands significant time and resources beyond their usual operations.
Remarkable Efficacy in Trials
The drug, which targets a genetic mutation present in approximately 90 percent of pancreatic cancers, demonstrated remarkable efficacy in trials. It extended median survival to 13.2 months, a significant improvement over the 6.7 months observed in patients receiving standard chemotherapy. "Doubling survival compared to best available chemotherapy is a big deal," commented Dr. Gulam Manji, co-director of the pancreas center at Columbia/New York-Presbyterian. "It is not a cure, but I think that this drug is a new breakthrough we can build on." Dr. Manji noted that in his decade-long career, he had only sought compassionate use for one other experimental drug, yet recently, seven patients inquired about daraxonrasib in a single day.
Complex Access Process
Gaining access to daraxonrasib is far more complex than obtaining a prescription for an FDA-approved medication. "Patients are already aware of the press release and are already calling," said Dr. Vincent Chung, a pancreas cancer specialist at City of Hope. "The challenge now is how to proceed." The process requires physicians to submit individual patient requests to Revolution Medicines. If deemed suitable by the company, these details must then be forwarded to the FDA, with hospital monitoring boards overseeing the patients. Dr. Chung expressed concern about the potential volume, stating, "Given the volume, I am not sure what will happen on the FDA side. I'm sure they don't want to have 10,000 applications at once," suggesting the agency might establish a more general enrollment protocol. While centers won't need to collect detailed data, they must report serious side effects.
Expedited Review and Public Attention
Daraxonrasib was among the first products accepted into the FDA's new expedited review process last year, potentially leading to approval within one to two months of a complete application submission. Revolution Medicines, based in Redwood City, California, did not immediately respond to a request for comment. CEO Mark Goldsmith, speaking on a recent conference call, declined to estimate the number of patients seeking early access or provide a timeline for a full FDA submission, though he affirmed, "there's a full-throttle effort to do it." The drug gained further public attention when former Nebraska U.S. Senator Ben Sasse revealed on "60 Minutes" that he has Stage 4 pancreatic cancer and is taking the Revolution drug.
The FDA did not respond to a request for comment regarding the program. The expedited voucher program was previously highlighted as a key achievement by Dr. Marty Makary, who resigned as FDA Commissioner after weeks of disagreements with administration officials. Pancreatic cancer remains a devastating disease, with the American Cancer Society estimating that around 67,000 people in the United States will be diagnosed this year, and 53,000 will succumb to it. "We are doing this as a service to our patients," Dr. Chung concluded. "I'm hoping of course that the FDA is going to review the data and then there is an approval much sooner than is typical."
