Major news for pancreatic cancer patients: The FDA, the U.S. Food and Drug Administration, approved daraxonrasib on Wednesday, a new targeted drug for patients with metastatic pancreatic cancer, six and a half months ahead of the target date set for completing its review.
The drug, taken as a pill once a day, is expected to become available for import to Israel soon, but its price could reach tens of thousands of shekels a month. In the video below, the audience rose to its feet and cheered when the study results were presented at the ASCO oncology conference in Chicago.
The audience rose to its feet as the daraxonrasib trial results were presented
(Video: From Mark Lewis’ X account)
“Pancreatic cancer is considered one of the more difficult cancers today, and the statistics have not changed very much in recent years,” said Prof. Ido Wolf, director of the oncology division at Tel Aviv Sourasky Medical Center, Ichilov Hospital. “The chance of someone with metastatic disease being cured is close to zero, and life expectancy has been roughly somewhere between a year and a year and a half. I’m talking only about metastatic disease, because that is what the drug is for. Once a person is diagnosed with metastatic pancreatic cancer, the statistics point to limited survival, and the treatment is difficult chemotherapy.”
About 80% to 90% of pancreatic tumors carry a mutation in a protein called RAS, which plays a central role in tumor growth.
“It is like the gas pedal driving the tumor forward,” Wolf said.
For decades, billions of dollars have been invested in attempts to develop drugs that inhibit the protein, without success. Only recently have drugs begun to emerge that can inhibit its mutated form, and daraxonrasib is the first to demonstrate effectiveness in a Phase 3 trial.
Pancreatic cancer is considered a silent and deadly form of the disease
(Photo: Shutterstock / MattL_Images)
“One important thing to remember is that this is the first drug, and we already know there will be more drugs in the future,” Wolf said. “This is the first generation, a breakthrough, and many good things are still ahead of us.”
One of the major challenges in developing drugs against RAS, he said, stems from the protein’s structure.
“There is no way to inhibit the protein directly because it is a ‘smooth’ protein,” Wolf said. “Anything you try to put on it just slides off.”
Daraxonrasib gets around that obstacle indirectly. The small molecule binds to an intracellular protein. Together, they form a structure that attaches to the active form of RAS and blocks the signaling that promotes tumor growth.
“Instead of locking it with a lock, it is as though they put a helmet on it and sealed it off,” Wolf said.
Median survival nearly double
The Phase 3 trial included 500 patients with metastatic pancreatic cancer who had already received previous treatment. Daraxonrasib was given as a single oral treatment at a dose of 300 milligrams once a day.
Median survival among patients who received the drug was 13.2 months, compared with 6.7 months among those who received standard chemotherapy.
Prof. Ido Wolf
Photo: Ryan Preuss“Adding half a year of life in this tumor is a real revolution,” Wolf said. “People will say, ‘Only half a year?’ The answer is simple: With lung cancer, the first inhibitors initially added half a year too, and look where we are today. There is great hope here for what comes next and for additional drugs. The excitement is not only about what we have today, but also about the future and the breakthrough. The very fact that science has managed to crack and inhibit this protein is an enormous achievement.”
The FDA approved the drug about six months ahead of the scheduled completion of its review after it was placed on a pathway intended to speed evaluation of treatments addressing significant unmet medical needs.
“That shows how strongly the FDA understood the significance and understood that you cannot leave these patients without a drug and that this had to move forward quickly,” Wolf said. “It is unusual. It shows how exceptional the drug is considered. We also have to remember that these patients have no other alternative.”
The less encouraging news: the price
Wolf said that once the drug is distributed in the United States, it will also be possible to import it to Israel.
“The import pathway is known, established and not overly complicated,” he said.
But the possibility of importing the drug raises another difficult question: Who will be able to afford it?
Until approval was granted, the company operated a compassionate-use program in the United States under which eligible patients could receive the drug free of charge.
“The company committed to providing it free until FDA approval, and only to patients in the United States,” Wolf said. “We already have patients whom we sent to the U.S. and who are receiving the drug there for free.”
Following the approval, the compassionate-use program is expected to close and the drug will be sold at full price.
The official price has not yet been published, but Wolf said estimates are around $30,000 a month, nearly 90,000 shekels.
“If that is indeed the price, it will be very complicated,” he said. “If we assume six months of treatment and round the cost to 100,000 shekels a month, we get to 600,000 shekels. Anyone with private insurance is in a better position.”
Public funding for the drug is also not expected in the near future. The list of drugs being considered for Israel’s 2027 health basket has already closed, meaning that unless the Health Ministry takes an exceptional step, the earliest the drug could be included is January 2028.
That would leave a gap of nearly a year and a half during which the drug could be available in Israel but without public funding.
“We will have patients who do not have private insurance and do not have the money, and for them there will be no drug,” Wolf said.
The early approval offers real hope to patients with few treatment options. But in Israel, the path to the drug does not end with medical approval. The next battle will be over who can receive it and who will be forced to wait.