The US Food and Drug Administration announced Wednesday it has approved a groundbreaking treatment option for pancreatic cancer.
Manufactured by Revolution Medicines, daraxonrasib made headlines earlier this year when former Nebraska Sen. Ben Sasse shared details about his experience with the drug, which left his skin bloody and “bubbling” but is also giving him more time with his family amid his battle with Stage 4 pancreatic cancer.
Clinical trial results showed that daraxonrasib, which will be sold under the brand name Rasonque, could double survival time for patients with pancreatic cancer, one of the deadliest cancers.
“This is life-altering,” Dr. Carla Kurkjian, an oncologist at Mercy Hospital in Oklahoma City, told the Wall Street Journal. “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.”
Patients taking the drug had a median overall survival of 13.2 months compared to 6.7 months for the chemotherapy group — a difference Dr. Nicholas Hornstein, a medical oncologist at Northwell’s Lenox Hill Hospital, called “remarkable.”
When trial findings were presented to cancer researchers last spring, the WSJ reports, they were met with a standing ovation.
Rasonque is a tablet taken orally once a day. It worked by targeting a mutated protein called KRAS, which Hornstein said acts like a “stuck accelerator” in cancer cells, telling them to keep growing when they should stop.
“Scientists have been trying to block KRAS for decades with little success,” Hornstein said.
“Recent drugs have managed to target one specific faulty version of KRAS, but daraxonrasib appears to work across multiple versions, which means it could potentially apply to far more patients — and far more cancer types, including colorectal and lung cancer.”
However, a relatively common side effect of the drug is skin toxicity, which Sasse experienced.
The 54-year-old father of three, who was diagnosed last year, was candid about his health battle and treatment in an interview with the New York Times in April.
“I take it orally, but it’s a nasty drug,” he said. “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.”
He added that his skin and face felt “nuclear.”
A pharmacist so taken aback by his appearance asked if 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.
At the time, he said his pain was down roughly 80% from where it started, even as he still deals with a bloody face and “strong waves of desire to puke.” The tumor volume in his torso was also down 76%, though he did not expect the drug to lead to a miraculous recovery.
Hornstein qualified that the toxicity is “generally far less than what Mr. Sasse reported.”
“Additionally, involving onco-dermatology early and using some newer agents, we are having significant success in mitigating these side effects,” he said.
In October, the FDA gave daraxonrasib one of the first vouchers in a new fast-track program aimed at getting promising drugs to patients faster than ever.
Acting FDA Commissioner Kyle Diamantas said Wednesday that the drug 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.”
Pancreatic cancer is resistant to many standard cancer treatments, making it notoriously difficult to treat. As a result, it is highly fatal, ranking as the third-leading cause of cancer deaths in the US.
Between 2015 and 2021, just 13.3% of people diagnosed with pancreatic cancer were still alive five years later, according to the National Cancer Institute.
In early trials for advanced pancreatic cancer, about one-third of patients taking daraxonrasib saw their tumors shrink, and most saw their cancer stabilize or improve.
When daraxonrasib was combined with standard chemo, more than half of patients had their tumors shrink, and 90% had their disease under control.
According to Revolution Medicines, their results have never been reported in any previous Phase 3 clinical trials for the disease.
“These striking results firmly support daraxonrasib as the new standard of care for patients with previously treated metastatic pancreatic cancer,” Dr. Mark Goldsmith, the company’s chief executive officer and chairman, said in a statement earlier this year.
“This is a monumental opportunity for patients and their families, and we need to do everything we can to clear the decks and make that happen as quickly as possible,” he added this week, according to WSJ.
