New cancer drug hailed as “life-altering” after FDA approval, but patients can face brutal side effects
A new treatment for one of the deadliest forms of cancer has just cleared a major hurdle in the United States, giving patients a new option in a disease that has long been notoriously difficult to treat.
The Food and Drug Administration has approved daraxonrasib, a daily pill developed by Revolution Medicines that will be marketed as Rasonque.
The approval comes after clinical trial results showed a striking difference in how long patients lived. Those receiving the drug had a median survival of 13.2 months. For patients treated with chemotherapy, the median was 6.7 months.
Dr. Nicholas Hornstein, a medical oncologist at Northwell’s Lenox Hill Hospital, called the gap “remarkable,” according to VT.
And for doctors who have spent years treating pancreatic cancer with limited options, the development could represent a major shift.
“This is life-altering,” Dr. Carla Kurkjian, an oncologist at Mercy Hospital in Oklahoma City, told the Wall Street Journal, according to the outlet.
“Not just for patients, as far as helping them extend their lives beyond anything we’d ever seen, but for us as physicians, to be able to have a different conversation than we’ve had for so long.”
Why researchers are so interested in Rasonque
The treatment takes aim at KRAS, a mutated protein that can essentially push cancer cells to continue multiplying. Hornstein has compared the protein to a “stuck accelerator” inside a cancer cell.
“Scientists have been trying to block KRAS for decades with little success,” he said.
Daraxonrasib could be different because it does not appear to be limited to just one form of KRAS. Instead, it can act against several versions of the mutated protein.
That broader activity could potentially allow the drug to help more patients. Researchers also believe the approach could eventually have a role in treating other cancers, including colorectal and lung cancer.
The treatment showed encouraging results even before its FDA approval.
Among people with advanced pancreatic cancer, around one in three patients taking daraxonrasib saw their tumors shrink. Most of the remaining patients experienced either stabilization or improvement in their disease.
Adding chemotherapy to the treatment produced even stronger results: more than half of patients saw their tumors shrink, while 90% had their disease controlled.
But there is another side to the drug – and it can be difficult to ignore.
The treatment can cause severe skin problems
Patients taking daraxonrasib can experience skin-related side effects, with former Nebraska Sen. Ben Sasse offering perhaps the most striking account of what that can look like.
Sasse, who is 54, was diagnosed with Stage 4 pancreatic cancer last year and has discussed his treatment publicly. He told the New York Times in April that the medication had caused severe problems with his skin.
“I take it orally, but it’s a nasty drug,” he told the New York Times in April. “It causes crazy stuff like my body can’t grow skin and so I bleed all out of a whole bunch of parts of me that shouldn’t be bleeding.”
Sasse described his face and skin as “nuclear.”
His appearance was reportedly so alarming on one occasion that a pharmacist asked whether doctors had done something “electrical” to him.
“I don’t even know what that is, but either acid or electric shocks produce a face that looks this hideous,” Sasse said with a laugh.
Yet despite the severity of the reaction, Sasse said the drug had also made a significant difference in his condition. He reported that his pain had decreased by roughly 80%. The volume of the tumor in his torso had also fallen by 76%. That does not mean the treatment has been easy.
Sasse was still experiencing a bloody face and “strong waves of desire to puke,” and he did not believe the drug would somehow lead to a miraculous recovery. Doctors have also emphasized that his reaction should not be viewed as typical for everyone taking the medication.
According to Hornstein, the toxicity associated with daraxonrasib is “generally far less than what Mr. Sasse reported”.
There are also ways doctors can work to reduce some of the treatment’s effects.
“Additionally, involving onco-dermatology early and using some newer agents, we are having significant success in mitigating these side effects,” he added.
A major development for pancreatic cancer patients
The FDA’s decision is particularly significant because pancreatic cancer remains exceptionally challenging to treat.The disease is resistant to many commonly used cancer treatments and is the third-leading cause of cancer deaths in the United States.
The five-year survival rate remains low. National Cancer Institute data show that only 13.3% of people diagnosed between 2015 and 2021 were still alive five years later.
The FDA had already moved daraxonrasib into a new fast-track program in October as part of an effort to make promising treatments available more quickly.
Acting FDA Commissioner Kyle Diamantas said the approval gives patients another option against a cancer that has historically been extremely difficult to treat. The approval “provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer.”
“It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible,” he added. “I am immensely proud of the dedicated FDA scientists whose fast, thorough review and relentless commitment made this groundbreaking milestone a reality.”
Dr. Mark Goldsmith, the CEO and chairman of Revolution Medicines, described the approval as a “monumental opportunity for patients and their families.”
And while researchers still have more to learn about the treatment, Dr. Shafia Rahman, a medical oncologist at The Ohio State University Comprehensive Cancer Center, believes the approval marks an important change for patients.
“This is not the end of the story, but a meaningful beginning,” he said. “For patients and families, this approval offers something we have not been able to offer before: a targeted therapy option that is both more effective and more tolerable.
“This is real, measurable hope that didn’t exist a year ago.”
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