By Deena Beasley and Nancy Lapid
May 14 (Reuters) – U.S. cancer centers are scrambling to enroll patients in an early access program for a highly promising pancreatic cancer drug from Revolution Medicines while they await what they hope will be a speedy FDA approval.
The Food and Drug Administration allowed the expanded access program on May 1, less than three weeks after Revolution said the once-daily pill, daraxonrasib, doubled survival in a clinical trial of patients with advanced pancreatic cancer, among the deadliest of cancers with one of the lowest 5-year survival rates.
The company had asked the FDA for permission to make it available free-of-charge for patients with previously treated pancreatic cancer that has spread to other parts of the body.
“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. “Cancer centers are all figuring out how to engage with our own institutions, opening up the protocols to provide access.”
Oncologists said getting approval and managing the expanded access program will take time and require cancer centers to dedicate substantial resources outside of their usual operations.
The drug was one of the first products accepted by the FDA last year for its new expedited review process, and could potentially get approval a month or two after a complete application is filed.
Revolution Medicines CEO Mark Goldsmith, speaking on a conference call last week, did not give a time frame for a full FDA submission. “There’s a full-throttle effort to do it,” he said.
The Redwood City, California-based company said it has already experienced high demand for the drug and expects it to remain high throughout the early-access program.
“We are actively supporting physicians through this process with safe, compliant, and rapid patient access as our top priority,” Revolution said in a statement.
“All requests must be initiated by a licensed treating physician and reviewed by an institutional review board,” the company said, adding that it expects to be able to respond to requests from physicians within two business days of receipt.
A BREAKTHROUGH TO BUILD ON
Former Nebraska U.S. Senator Ben Sasse recently revealed that he has Stage 4 pancreatic cancer and told the CBS news program “60 Minutes” that he is taking the Revolution drug.
The drug, which targets a genetic mutation found in about 90% of pancreatic cancers, was shown in a clinical trial to extend median survival to 13.2 months compared with 6.7 months for patients on chemotherapy.
“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. “It is not a cure, but I think that this drug is a new breakthrough we can build on.”
In a 10-year career, Manji could recall seeking compassionate use of an experimental drug for just one other patient. On a recent day in the clinic, the Columbia oncologist said seven patients asked him about starting treatment with daraxonrasib.
Getting them access is not as simple as writing a prescription for an FDA-approved drug, he and other cancer specialists said.
“Patients are already aware of the press release and are already calling,” said Dr. Vincent Chung, pancreas cancer specialist at City of Hope. “The challenge now is how to proceed.”
The program requires physicians to submit requests for each individual patient to Revolution Medicines, Chung said, and if the company decides they are a good candidate, all those details then need to be submitted to the FDA. Hospital monitoring boards will need to follow the patients.
“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, adding that the agency may instead set up 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, but will need to report serious side effects or other issues.
The FDA did not respond to a request for comment. The expedited voucher program was touted as a signature achievement by Dr. Marty Makary, who resigned as FDA Commissioner on Tuesday after weeks of clashes with Trump administration officials.
Around 67,000 people in the United States will be diagnosed with pancreatic cancer this year, and 53,000 will die of the disease, according to the American Cancer Society.
“We are doing this as a service to our patients,” Chung said. “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.”
(Reporting By Deena Beasley in Los Angeles and Nancy Lapid in Tucson; additional reporting by Julie Steenhuysen in Chicago; editing by Caroline Humer and Bill Berkrot)
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