FDA approves ‘landmark’ pancreatic cancer drug that nearly doubles patients’ survival

Patients battling one of medicine's toughest cancers now have a new weapon, after regulators gave the green light this week to a drug oncologists are already calling transformative.

The Food and Drug Administration has signed off on Rasonque, known chemically as daraxonrasib, which is a daily pill developed by Revolution Medicines for people with advanced pancreatic cancer who've already been through at least one prior treatment, such as chemotherapy.

The approval hinges on results that stunned even the doctors running the trial.

Among 500 patients tracked in a late-stage study, those given Rasonque alongside standard chemo survived for an average of 13.2 months - roughly double the survival time of patients who received chemo on its own, according to NBC News, which first reported the approval.

For a disease with such grim odds, that kind of jump has left specialists struggling to find comparisons.

Dr Rachna Shroff, who leads the division of haematology and oncology at the University of Arizona Cancer Center in Tucson, told NBC she broke down when she first saw the trial's early data back in April.

'It's that big of a game changer for those of us who treat pancreatic cancer,' she said. 'It's unprecedented.'

Wednesday's approval comes several months after those trial results first began circulating within the oncology community back in April, when Shroff and others reviewed the preliminary data.

The drug is taken as a once-daily pill and targets a mutation in the KRAS gene, which is present in nearly all pancreatic cancers. (Getty stock image)

How does Rasonque actually work?

Taken once a day as a tablet, Rasonque zeroes in on a fault in a gene called KRAS - a mutation present in almost every case of pancreatic cancer, and also common in lung, colorectal and ovarian tumours.

Under normal circumstances, KRAS behaves like a switch that tells cells when to grow.

The mutated version gets locked in place, letting cancer cells multiply unchecked.

Researchers have spent years trying to build a drug capable of flipping that switch back off, and past efforts to do so had repeatedly come up short - until Rasonque.

Channing Der, a pharmacologist at the University of North Carolina at Chapel Hill, described the approval as a "monumental moment for the treatment of pancreatic cancer" in emailed comments.

He added that pancreatic cancer is 'the third leading cause of cancer death in the U.S. and the deadliest of all cancers,' calling this breakthrough 'just the beginning of more advances to come.'

That severity is exactly why a new option matters so much. The American Cancer Society projects over 67,000 new pancreatic cancer diagnoses across the US this year.

There's currently no screening test for the disease, and symptoms in the early stages are frequently too subtle to catch it before it spreads - a major reason why, according to official figures, just eight percent of patients with pancreatic adenocarcinoma, the disease's most common form, live for five years or longer after diagnosis.

The FDA have approved a drug to tackle pancreatic cancer that brought one researcher to tears when she read the results (Getty stock image)

Does Rasonque come with side effects?

The drug isn't free of drawbacks. Documented side effects include a blistering rash, sores across the mouth and throat, plus vomiting and diarrhoea.

Former Nebraska Senator Ben Sasse, who took Rasonque as part of a clinical trial, spoke about the rash on a New York Times podcast earlier this year, branding it 'nuclear.'

The company says over 2,000 people have accessed Rasonque already, through an early-access scheme it rolled out back in May, ahead of this week's formal sign-off.

Revolution Medicines has yet to confirm how much a course of the drug will cost patients.

UNILAD has contacted Revolution Medicines for comment.