Cancer treatment centers nationwide are mobilizing to sign up patients for an early access program for daraxonrasib, an experimental once-daily pill from Revolution Medicines, after the Food and Drug Administration permitted an expanded access pathway on May 1. The move came less than three weeks after the company announced clinical trial results in which the drug doubled median survival for patients with advanced pancreatic cancer compared with chemotherapy.
Revolution Medicines asked the FDA to allow the medicine to be provided free-of-charge to patients whose pancreatic cancer had previously been treated and had spread to other parts of the body. The company is based in Redwood City, California, and did not immediately respond to a request for comment.
Immediate patient demand and institutional response
Physicians and hospital programs report a surge of inquiries following the FDA announcement. "The public caught wind of the FDA announcement... which has triggered a deluge of patient requests," said Dr. Daniel King, medical oncologist at the Zuckerberg Cancer Center of Northwell Health. He added that cancer centers are working to align internal processes and open protocols to provide access.
Oncologists caution that enrolling patients through an expanded access program is more involved than prescribing an approved medication. A Columbia/New York-Presbyterian pancreas specialist said multiple patients have already asked about starting treatment. "Doubling survival compared to best available chemotherapy is a big deal," said Dr. Gulam Manji, co-director of the pancreas center at Columbia/New York-Presbyterian. He emphasized the significance of the survival result while noting that the therapy is not a cure: "It is not a cure, but I think that this drug is a new breakthrough we can build on." Over a 10-year span, Manji said he could recall seeking compassionate use of an experimental drug for only one other patient; on a recent clinic day he said seven patients asked him about daraxonrasib.
Operational and regulatory steps required
Physicians must submit individual patient requests to Revolution Medicines, according to clinicians involved in the process. If the company determines a patient is an appropriate candidate, the next step requires submission of details to the FDA. Hospital institutional review or monitoring boards will also be responsible for following patients enrolled in the program.
"The challenge now is how to proceed," said Dr. Vincent Chung, pancreas cancer specialist at City of Hope. He noted the volume of prospective participants could present a bottleneck for the FDA: "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," Chung said, and suggested the agency might instead establish a more general enrollment protocol.
Manji said his understanding is that cancer centers will not be required to collect detailed data on patients treated under the expanded access program, although they will need to report serious side effects or other significant issues.
Clinical results and target population
In the trial cited by Revolution Medicines, daraxonrasib extended median survival to 13.2 months, compared with 6.7 months for patients receiving chemotherapy. The drug targets a genetic mutation present in about 90% of pancreatic cancers.
Revolution included the drug as one of the first products accepted into the FDA's new expedited review process last year, raising the possibility of approval a month or two after a complete application is filed. Speaking on a conference call last week, CEO Mark Goldsmith declined to estimate how many patients would seek early access or to provide a firm timeline for a full FDA submission, saying only, "There’s a full-throttle effort to do it."
Among public figures seeking the drug, former Nebraska U.S. Senator Ben Sasse recently disclosed he has Stage 4 pancreatic cancer and informed the CBS news program "60 Minutes" that he is taking the Revolution drug.
Regulatory context and mortality statistics
The FDA's expedited voucher program has been highlighted by some officials; the program was described in the context of the resignation of Dr. Marty Makary, who resigned as FDA Commissioner on Tuesday after weeks of clashes with Trump administration officials. The agency did not respond to a request for comment about the expanded access program.
Pancreatic cancer remains one of the deadliest cancers. According to figures cited from the American Cancer Society, about 67,000 people in the United States will be diagnosed with pancreatic cancer this year and about 53,000 will die from the disease.
What oncologists say now
Clinicians involved with patient care stressed that participation in the expanded access program is being pursued primarily as a service to patients facing a disease with poor outcomes. "We are doing this as a service to our patients," Chung said. He added that physicians hope the FDA will evaluate the trial data and move to approval "much sooner than is typical," though no formal approval timeline has been provided.
The combination of strong trial results, high patient interest and operational complexity leaves hospitals and regulators balancing rapid access against the need to follow procedural and safety steps. For centers and specialists, executing the expanded access program will require dedicating resources beyond routine clinical workflows while they wait for the company to complete any full regulatory submission and for the FDA to act.