The Food and Drug Administration approved on Wednesday a new drug for advanced pancreatic cancer, ushering in a new era of treatment for a disease that has long been considered one of the most hopeless in medicine.
The drug, taken as two pills a day, is the first of its type and has electrified cancer specialists and patients. It is not a cure, but it is the first treatment to substantially extend the lives of patients with pancreatic cancer.
The drug, daraxonrasib, is made by Revolution Medicines and will be sold under the brand name Rasonque. It was approved for patients who have metastatic pancreatic cancer and have already tried chemotherapy. In a key clinical trial, participants who got daraxonrasib lived for a median of over 13 months, compared with less than seven months for those who got chemotherapy. Some patients who have gotten the drug through clinical trials have lived for years.
“We’ve never seen a benefit like this,” said Dr. Anna Berkenblit, the chief scientific and medical officer at the Pancreatic Cancer Action Network, an advocacy group for research and patients.
Revolution Medicines did not immediately announce how much it planned to charge for the drug. Wall Street analysts have said they expect the drug’s sticker price to be several hundreds of thousands of dollars a year. The vast majority of the cost is expected to be covered through insurance, with varying out-of-pocket costs for patients.
Since May, more than 2,000 patients have received free, early access to the drug through an expanded access program that the company set up after its key clinical trial concluded. The company said that people receiving the drug through this pathway would transition to getting it covered through insurance.
The pancreas, a gland deep in the abdomen, helps regulate blood sugar and digestion. Pancreatic cancer kills more than 50,000 people in the United States a year. Only 3 percent of patients whose pancreatic cancer has spread to distant parts of their body live for five years after being diagnosed. The few available treatment options, typically grueling chemotherapy, do little to extend life. For years researchers had despaired of even finding a more effective treatment for this cancer.
So at a cancer meeting in May, oncologists stood up and applauded — and, according to people in the room, some wept — when the clinical trial data was presented showing a doubling in survival times.
The researcher who gave that presentation and led the key study, Dr. Brian Wolpin of the Dana-Farber Cancer Institute in Boston, said in an interview that he was stunned by the trial results when he was finally allowed to see them.
“I have not seen anything like that before in trials we have run in pancreatic cancer,” he said. “I just kept repeating, ‘Wow.’”
Nearly all pancreatic cancers, as well as many lung and colon cancers, are fed by a mutated cellular protein called KRAS. Without it they die. But there seemed to be no toehold for a drug to attack the smooth-surfaced KRAS protein and block it. For that reason, the longstanding conventional wisdom in the field was that it would be impossible to develop a drug that homes in on KRAS.
But over decades, a series of scientific advances upended that dogma. Daraxonrasib’s success showed that KRAS could be conquered. It uses a novel approach that glues molecules together to grab and shut down KRAS.
Daraxonrasib often causes side effects like rash, diarrhea, fatigue and nausea. Several patients who took the drug in clinical trials said that the side effects could be harsh. Some said that extending lives by 13 months was not enough — they wanted years, decades even, with their families.
But over the past few years, daraxonrasib studies began reporting such promising early results that patients around the world scrambled for spots in clinical trials that were testing it.
Dr. Nilofer Azad, a pancreatic specialist at Johns Hopkins, said in an interview this spring that she was getting emails every day from people around the world who were trying to get into a study to receive the drug.
The most high-profile clinical trial participant on the drug is former Senator Ben Sasse of Nebraska, who announced last December that he had been diagnosed with metastatic pancreatic cancer. He has credited the drug with extending his life, saying that tumor markers in his blood have plummeted since he started it.
Oncologists say daraxsonrasib is only the beginning of what they see as a new era in treating pancreatic cancer and other cancers fueled by KRAS. Revolution Medicines and other companies are testing dozens of similar KRAS drugs in clinical trials. The drugs are being tested alone, in combination and along with chemotherapy and immunotherapy. They also are being tested as initial therapies for pancreatic cancer.
The hope now is that as treatments evolve and improve, most patients might be able to keep their cancers in check for years, or even indefinitely.
Yet so many questions remain, said Dr. Mark Goldsmith, the chief executive at Revolution Medicines, in a recent webinar.
“Why do tumors take so much time to shrink?” he asked. “Why don’t they shrink instantly? What’s left after a tumor is getting crushed by a drug?”
And why are some rare patients able to clear the tumors from their bodies when they take daraxsonrasib?
One such patient is Jerry Simonson, 79, who lives near Salt Lake City.
He found out he had pancreatic cancer after seeing a doctor in 2017 for a shoulder infection. The cancer had already spread to his lymph nodes.
Mr. Simonson thought he was doomed. “I just said to myself, ‘Two months,’” he recalled. “I knew two other people when they were diagnosed. They only had two months left.”
But he enrolled in a clinical trial of daraxsonrasib in late 2022, and his tumor started to shrink. Today, it cannot be seen on scans. He says he feels fine — he walks two miles every day and is learning to play pickleball. His side effects have been minor — a mild rash on his face and chest that he controlled with a steroid cream, and some constipation and diarrhea.
He added that he didn’t realize until recently how revolutionary his drug was. “It was just another doctor, another pill,” Mr. Simonson said.
Then, in May, he was watching television and saw coverage of the cancer meeting where oncologists gave daraxsonrasib a standing ovation.
“That’s when it really hit home,” Mr. Simonson said.
