FDA approves groundbreaking new pancreatic cancer drug

The Food and Drug Administration (FDA) on Wednesday approved a treatment for advanced pancreatic cancer, offering new life-extending care for patients with one of the most aggressive forms of cancer.

The once-daily pill, called daraxonrasib and manufactured by Revolution Medicines , will be sold under the brand name Rasonque. It was approved for patients who already tried one line of therapy, or if they are not candidates for initial chemotherapy.

The drug is not a cure. But it’s the first real option for extending the life of patients diagnosed with pancreatic cancer.

Pancreatic cancer accounts for a disproportionately high share of cancer deaths, despite representing only about 3 percent of all cancer diagnoses. The aggressive disease is typically detected late, and there have been historically limited treatment options that do little to extend a patient’s life. The five-year survival rate is only about 8 percent.

“The FDA approval of Rasonque is a monumental step forward for patients with pancreatic cancer and for the oncology field,” said Mark Goldsmith, chief executive officer and chair of Revolution Medicines, in a statement.

“For the first time, patients have an approved targeted medicine designed to directly inhibit the main cause of pancreatic cancer, RAS, which has been one of the most intractable disease targets since its discovery decades ago,” Goldsmith said.

Daraxonrasib attacks the underlying genetic causes of pancreatic cancer. It targets a group of proteins called RAS, which when mutated are a key driver of tumor growth in most patients with pancreatic adenocarcinoma.

Patients with advanced pancreatic cancer who received the drug during a key clinical trial saw a median overall survival of 13 months, compared to just six months for patients who were offered standard chemotherapy.

The trial found daraxonrasib reduced the risk of death by 60 percent compared with chemotherapy.

“Today’s 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,” acting FDA Commissioner Kyle Diamantas said in a statement, adding that the approval was a “groundbreaking milestone.”

Revolution Medicines did not immediately announce how much it planned to charge for the drug.

The FDA granted Rasonque a priority review voucher, which is intended to help accelerate the review of therapies that address national public health priorities.

The company opened an FDA-cleared expanded access program in May and has since distributed the drug to more than 2,000 patients through participating academic cancer centers and community oncology practices.

The most common adverse reactions in patients were rash, diarrhea, stomatitis, nausea, vomiting, abdominal pain, edema, decreased appetite and hemorrhage.

One of the most high-profile clinical trial participants is former Sen. Ben Sasse (R-Neb.), who announced in December he had been diagnosed with Stage 4 pancreatic cancer. Sasse told The New York Times in April that he credits the drug with shrinking his tumors, but the cancer has likely spread to so many other parts of his body “that it’s probably just not something you can ever catch up on.”