FDA approves once-daily pill for advanced pancreatic cancer

The U.S. Food and Drug Administration approved a once-daily pill for advanced pancreatic cancer, authorizing its use for adults with metastatic disease after at least one prior treatment or when certain combination therapies could not be given, the agency said Wednesday.

FDA approval extends access to Rasonque, manufactured by Revolution Medicines in California, which is designed to inhibit a protein network driven by RAS and taken once daily.

In an FDA press release, the agency said it cleared the drug more than six months ahead of its review deadline and described it as a new treatment option for patients with advanced pancreatic cancer.

"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," said Acting FDA Commissioner Kyle Diamantas, J.D.

The authorization covers adults with metastatic pancreatic cancer that either did not respond to at least one prior treatment or could not receive certain combination drug therapies, according to the FDA.

Pancreatic adenocarcinoma accounts for up to 95% of roughly 67,000 annual pancreatic cancer cases identified by the National Cancer Institute, and it makes up 3.2% of total cancer diagnoses while causing a disproportionately high share of cancer deaths.

Scientific American and Fox News reported that approval followed a randomized open-label trial of 500 patients with pre-treated metastatic pancreatic disease.

In the trial, median overall survival was 13.2 months for patients taking the pill, compared with 6.7 months in the standard chemotherapy control group, as described by FDA.

"This drug showed unprecedented results in an area of high unmet need," said Angelo de Claro, M.D., director of the FDA’s Oncology Center of Excellence.

The FDA said it accelerated the medication’s path to market through Priority Review, Orphan Drug status, and a Breakthrough Therapy designation, and used the FDA commissioner’s National Priority Voucher pilot program to speed access to critical public health treatments.

Before final approval, the agency issued a "safe to proceed" letter in May, opening an expanded access protocol that allowed patients to receive the drug prior to the official green light.

Safety data cited by the FDA showed the most frequent adverse events included rash, diarrhea, mouth sores (stomatitis), nausea, fatigue, vomiting, abdominal pain, fluid retention (edema), reduced appetite, and bleeding (hemorrhage).

"Today’s landmark approval is the most significant advance we have seen in the fight against pancreatic cancer, a devastating disease," said Anna Berkenblit, M.D., chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN).

"I believe this drug will transform how pancreatic cancer is treated, giving people the opportunity for more time with loved ones, the possibility of a better quality of life, and optimism that continued research may lead to even greater advances," Berkenblit added.

Separately, WCVB reported that Dr. Anasuya Gunturi, chief of oncology at Lowell General Hospital, which is part of Tufts Medicine, said doctors could start ordering the FDA-approved drug for patients.

"It was pretty remarkable because metastatic pancreatic cancer is one of the most difficult cancers that we treat," said Dr. Anasuya Gunturi.

WCVB said Gunturi added that very few patients typically survive more than a couple of years and that prognosis is dismal, while the trial’s initial results were described as promising.