New hope for pancreatic cancer patients after FDA approves drug shown to improve survival
American patients facing advanced pancreatic cancer have been offered a more effective treatment after the Food and Drug Administration granted approval on Wednesday to a groundbreaking new therapy against the deadly disease.
Regulators gave expedited authorization to the novel pill, called daraxonrasib, which blocks a mutated protein driving tumor growth in over 90% of pancreatic cancer cases, a scientific approach that had previously eluded pharmaceutical researchers for decades.
Revolution Medicines will market the daily treatment under the brand name Rasonque.
Participants taking the drug almost doubled their survival time with fewer severe side effects in a trial randomly assigning them to receive the experimental pill or chemotherapy.
The company-funded study evaluated 500 individuals whose spreading, metastatic cancer no longer responded to prior treatment.
Patients on the new regimen lived for a median of 13.2 months compared to 6.7 months for chemotherapy recipients.
Side effects included skin rash, mouth sores, diarrhea, and digestive issues.
“This is not a cure, it’s one more option for these patients,” said Dr. Pashtoon Kasi of City of Hope Orange County, a California-based cancer center. “But it’s the best option we’ve ever had.”
Pancreatic cancer is among the deadliest forms largely because detection is difficult before malignancy spreads to other organs.
The American Cancer Society estimates roughly 67,000 new cases will be diagnosed in the United States this year, with over 52,000 deaths from the disease.
The overall five-year survival rate stands at 13%.
The therapy gained widespread public attention earlier this year after former Sen. Ben Sasse, R-Neb., detailed on CBS’ “60 Minutes” how he experienced less pain while taking it. Heightened interest led the FDA to permit “expanded access” prior to official clearance for patients meeting specific criteria.
FDA regulators noted on Wednesday that clearance was granted more than six months ahead of their target date.
“It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible,” the FDA’s acting commissioner, Kyle Diamantas, said in a statement.
Unlike other cancers benefiting from diverse chemotherapy alternatives, treating pancreatic cancer has proven far more difficult to tackle.
The novel therapy targets mutations in the RAS gene family regulating cell growth. Crucial KRAS mutations fuel pancreatic cancer growth, but a complex structure making it difficult for drugs to attach to mutated proteins meant this disease driver was long deemed “undruggable.”
Physicians treating pancreatic cancer hope the medication could usher in new treatments for other malignancies, with dozens of experimental drugs currently in development.
“I think this has opened doors for many other companies,” Kasi said. “Downstream I think there are going to be a lot more trials looking at this approach in other tumor types.”
Revolution Medicines’ therapy relies on what is essentially a molecular glue binding multiple KRAS subtypes. The Redwood City, California-based manufacturer is studying the technology across other cancers, including lung cancer.
