The Food and Drug Administration said Friday it would allow some patients with deadly pancreatic cancer to receive early access to a promising and highly sought-after drug.
The treatment, daraxonrasib, which is taken as three pills a day, is not yet approved for use. But many patients were desperate to try it. The drug recently generated the most encouraging clinical trial results ever seen in the pancreatic cancer field.
Revolution Medicines, the Silicon Valley company developing the drug, has requested permission to offer it to patients under a regulatory pathway known as expanded access. So far, no one has received the experimental drug outside of clinical trials, where patient demand far exceeds the limited number of spots, according to the company.
The FDA said patients with metastatic pancreatic cancer who have been “previously treated” would be eligible for the program. It was not immediately clear how soon patients could start taking the drug if they qualify. The company said it would provide the drug through the program at no cost to patients.
Pancreatic cancer (the gland buried deep in the abdomen that is involved in digestion and blood sugar regulation) has one of the grimmest prognoses in oncology, with many patients dying within months of diagnosis. The disease kills more than 50,000 Americans a year, accounting for about 8 percent of cancer deaths in the United States.
The few treatment options available often help little. Only 3 percent of people whose pancreatic cancer has spread to distant parts of the body live five years after being diagnosed.
Many patients and families have called for daraxonrasib to be offered through expanded access, saying they do not have time to wait for the drug to gain regulatory approval. The Trump administration has designated the drug for expedited review and could approve it later this year.
“We're in a situation where Ian could be dead before this comes to market,” said Emily Solari, whose husband, Ian Spradlin, 46, has metastatic pancreatic cancer.
Spradlin, who has already tried three lines of chemotherapy and two experimental drugs, said in an interview last weekend that he would be eager to try daraxonrasib with expanded access if he could get it that way.
“I want to spend more time with my children,” he said.
Expanded access, also sometimes known as compassionate use, aims to close the gap of months or years between the time a drug shows promise in studies and the time it is available on the market. It is intended for the sickest and most desperate patients who do not have good standard treatment options. Typically, the company that develops the drug provides it for free.
The FDA receives hundreds of early access requests annually. Doctors can request it for individual patients if a drug manufacturer agrees to provide the drug. The FDA said it granted more than 99 percent of these requests. Or, as with daraxonrasib, expanded access programs can be run for larger groups of patients.
In recent months, the FDA has come under fire from patient groups and doctors who were discouraged by delays and some denials in approving drugs, largely for rare diseases. Dr. Marty Makary, the agency's commissioner, responded by promising faster reviews for some drugs.
Last month, Revolution captivated the industry when it announced that daraxonrasib doubled survival time in a late-stage clinical trial. People who received the drug lived more than 13 months, compared to less than seven months for those who received chemotherapy, the company said in a news release.
The study included 501 patients with metastatic pancreatic cancer who had already tried a line of chemotherapy. A six-month increase in life expectancy is unheard of for people in that phase of the disease.
The researchers plan to present the data later this month at a cancer conference in Chicago. The findings have not yet been published in a peer-reviewed medical journal.
Daraxonrasib usually causes side effects such as rash, diarrhea, fatigue, and nausea. Several patients taking the drug in clinical trials said side effects, including raw and split fingertips, could be severe.
One prominent participant in the drug's clinical trials is Ben Sasse, a former Republican senator from Nebraska who revealed he had pancreatic cancer late last year. In a recent video interview in The New York Times Opinion, he attributed a rash on his face to a side effect of the treatment. He said the drug had shrunk his tumors and allowed him to reduce his use of painkillers.
In an interview this week, Dr. Mark Goldsmith, Revolution's chief executive, said the company had been responding to a flood of inquiries from patients asking for the drug. He said whenever clinical trial slots opened up for the drug, they were often secured on the same day.
“Demand for daraxonrasib has been very high for several years and it only increases every time we show data,” said Dr. Goldsmith.
“It is a desperate situation because it is a relentless disease,” he said. “What we can do about this is act quickly.”
Daraxonrasib targets a mutated protein known as KRAS that drives the growth of nearly all pancreatic cancers, as well as many lung and colon cancers. For a long time, some researchers thought it was impossible to develop a drug that targets KRAS.
Revolution, along with several other companies, is testing other drugs in earlier-stage clinical trials that work similarly to daraxonrasib. But there are not enough study places to accommodate what pancreatic cancer specialists described as an avalanche of patient requests. They said they hated telling patients that daraxonrasib was virtually unavailable.
“We have a huge waiting list,” said Dr. Nilofer Azad, a pancreatic cancer specialist at Johns Hopkins Medicine.
“I receive emails daily from people all over the world,” Dr. Azad said. “There are people who come here from abroad just so we can meet them,” he added.
Promising data on daraxonrasib has only increased patient demand.
On Wednesday, Raven Zachary, 52, of Portland, Oregon, met with an oncologist for the first time after receiving a preliminary diagnosis of metastatic pancreatic cancer. He brought with him a printout of his 19 most pressing questions.
The question at the top of your list: “What about daraxonrasib? Am I eligible based on my condition and do I have access?”
In interviews, patients and their families said they understood that daraxonrasib was not a cure. They said they had realistic hopes for what it could give them: more months with their families or the chance to travel without being tied to a chemotherapy infusion chair every two weeks. They said they were willing to accept the risk of side effects or that it might not work as well as the clinical trial results suggested.
“Every month that medication is delayed means I have to have chemotherapy twice more,” said Dr. Ronald Silvestri, 77, a retired pulmonologist in Natick, Massachusetts. He has metastatic pancreatic cancer and has already received 32 rounds of chemotherapy while trying unsuccessfully to participate in a clinical trial. “And it's getting harder and harder for me to tolerate it.”
Paths for desperate patients to obtain unapproved drugs have existed for decades and have sometimes sparked fierce battles over how quickly the drugs should be made available and to whom. The process was formalized in the 1980s, when dying AIDS patients lobbied for early access to treatments.
In 2021, patients with amyotrophic lateral sclerosis, or ALS, pressured Biogen, the company developing a drug for a rare genetic form of the neurodegenerative disease, to offer it through expanded access. The patient at the center of the campaign, Lisa Stockman Mauriello, was excluded from a clinical trial because of the date she had been diagnosed. But he finally received the drug through expanded access just days before he died and nearly two years before it won FDA approval.
Today, the daraxonrasib case has all the ingredients — a deadly disease, a lack of decent treatment options for patients, and clear data that the drug works — that “portray an ethical breach on the part of the company in establishing one of these programs,” said Holly Fernández Lynch, a bioethicist at the University of Pennsylvania.
FDA approval applies only at the national level; Patient requests must be made by a licensed physician in the United States. At least for now, that restriction will prohibit most desperate patients from other countries from getting the drug.
Cynthia Ataefe, 66, from Buenos Aires, has metastatic pancreatic cancer and wants to try the drug. But she is too sick to travel to attend a clinical trial, even if she could participate in one, according to her daughter, Cecilia Gerson, who lives in New York.
Gerson said earlier this week that the family was pinning their hopes on greater access. “I still hope that somehow we will get the medicine in time,” he said.
