FDA Approves New Pill for Advanced Pancreatic Cancer

The Food and Drug Administration (FDA) has granted approval for a new medication aimed at treating advanced pancreatic cancer. This innovative drug offers U.S. patients a more potent option against one of the most lethal cancer types.

Expedited Approval of Daraxonrasib

The FDA gave fast-tracked approval to daraxonrasib, a pioneering pill that targets a mutated protein driving tumor growth in over 90% of pancreatic cancer cases. This approach remained elusive to drugmakers for decades.

Revolution Medicines is marketing the pills under the brand name Rasonque, with a one-month supply priced at about $39,800.

Improved Survival Outcomes

A company-backed study showed patients on the drug nearly doubled their survival time with fewer severe side effects compared to chemotherapy. The study involved 500 patients whose cancer had spread and was unresponsive to previous treatments.

Dr. Céline Gounder from CBS News explained, “Most patients receive a pancreatic cancer diagnosis late, when it has already metastasized.” She added, “This pill inhibits a protein called RAS, which mutations stimulate in about 90% of all pancreatic cancers. Scientists have deemed RAS ‘undruggable’ for the last 40 years, yet we now have a drug that targets it.”

Patients on the new treatment lived for a median of 13.2 months versus 6.7 months for those receiving chemotherapy. Side effects included skin rash, mouth sores, diarrhea, and other digestive problems.

A Significant Advancement

Dr. Andrew Coveler of the Fred Hutch Cancer Center remarked, “Though this medicine isn’t a cure, it’s a notable advancement.” Pancreatic cancer ranks among the deadliest forms because it usually evades detection until it spreads to other organs.

The American Cancer Society estimates around 67,000 new cases will be diagnosed in the U.S. this year, and over 52,000 people will succumb to the disease. The five-year survival rate stands at a mere 13%.

Clinical Trial Testimonies

Former Senator Ben Sasse of Nebraska, diagnosed with stage-four pancreatic cancer last December, participated in the clinical trial for daraxonrasib. He described the drug’s effect on him as “miraculous” and noted a significant reduction in tumor volume.

Sasse shared with “60 Minutes” that upon diagnosis, doctors gave him “three to four months” to live. “I am on extended time already,” he stated.

FDA regulators highlighted that the drug’s approval came over six months ahead of schedule. Acting commissioner Kyle Diamantas emphasized, “We must provide cures and meaningful treatments to patients swiftly.”

Targeting RAS Gene Mutations

Unlike many other cancers benefiting from chemotherapy alternatives, pancreatic cancer poses challenges in treatment. The new drug targets mutations in the RAS gene family, crucial in regulating cell growth. Specifically, KRAS mutations are pivotal in pancreatic cancer’s progression.

A structure hindering drug attachment to these mutated proteins deemed this cancer driver “undruggable” for years. The new pill employs molecular glue to bind multiple KRAS subtypes.

Revolution Medicines, based in Redwood City, California, is testing their technology for other cancers, including lung cancer. Dr. Pashtoon Kasi of City of Hope Orange County noted, “This has set a precedent for numerous companies. More clinical trials in other tumor types are expected.”