Pancreatic cancer: FDA approves drug that targets a protein that was “undruggable” for decades

The U.S. Food and Drug Administration (FDA) approved a groundbreaking new drug for the most common form of pancreatic cancer on Wednesday, offering patients in the United States a more effective treatment against one of the deadliest types of cancer.

FDA authorities granted accelerated approval to the first-of-its-kind pill, called daraxonrasib, which blocks a mutated protein that fuels tumor growth in more than 90% of pancreatic cancer cases. It is an approach that had eluded drugmakers for decades.

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The daily pills will be sold by Revolution Medicines under the brand name Rasonque.

Patients who took the drug nearly doubled their survival time, with fewer serious side effects, in a study where they were randomly assigned to the experimental treatment or further chemotherapy. The study, funded by the company, involved 500 patients whose metastatic, or spreading, cancer had stopped responding to previous treatment.

Patients who received the new treatment lived for a median of 13.2 months, compared to 6.7 months for those who received chemotherapy.

“It is not a cure, but it is one more option for these patients,” said Dr. Pashtoon Kasi of City of Hope Orange County, a cancer care center based in California. “But it is the best option we have had.”

Pancreatic cancer is one of the deadliest forms of the disease, largely because it is difficult to detect before it begins to spread to other organs. The American Cancer Society estimates that about 67,000 new cases will be diagnosed in the United States this year and that more than 52,000 people will die from the disease. The overall five-year survival rate is 13%.

The drug attracted public attention earlier this year when former Republican Senator for Nebraska, Ben Sasse, described on CBS’s “60 Minutes” how he had experienced less pain while taking it. An increase in public interest led the FDA to allow “expanded access,” prior to the official approval of the medication, for patients who met certain criteria.

FDA regulators noted on Wednesday that they approved the drug more than six months ahead of its target date.

“It is our fundamental duty to bring more cures and meaningful treatments to patients as quickly as possible,” said Kyle Diamantas, acting FDA commissioner, in a statement.

Doctors who treat pancreatic cancer have expressed optimism that the approval of the drug could pave the way for more new options, with dozens of experimental medications in development. Unlike other cancers that have benefited from a variety of alternatives to chemotherapy, pancreatic cancer has been more difficult to address.

How does the new pill for pancreatic cancer work?

The new drug targets mutations in the RAS family of genes, which normally regulates cell growth. So-called KRAS mutations are especially critical in driving pancreatic cancer. But a structure that made it difficult for drugs to stick to the mutated proteins meant this cancer driver was long considered “undruggable.”

“I think it has opened the doors for many other companies,” Kasi said. “I think that, in the future, there will be many more trials looking at this approach in other tumor types.”

The Revolution Medicines drug essentially uses a molecular glue to bind to multiple KRAS subtypes. The pharmaceutical company, based in Redwood City, California, is also studying its technology in other forms of cancer, including lung cancer.