FDA approves once-daily pill for deadly cancer after patients' survival rates nearly double

The U.S. Food and Drug Administration approved the use of a new cancer treatment pill Wednesday.

Rasonque, manufactured by Revolution Medicines in California, is an inhibitor for the most common type of pancreatic cancer. The tablet, which is taken once daily, targets multiple forms of a protein called RAS that drives tumor growth.

In a press release, the FDA called Rasonque a "new treatment option for patients with advanced pancreatic cancer," noting the agency approved it more than six months ahead of its review deadline.

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"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 press release.

"I am immensely proud of the dedicated FDA scientists whose fast, thorough review and relentless commitment made this groundbreaking milestone a reality."

The authorization is for adults with metastatic pancreatic cancer that either did not respond to at least one prior treatment or could not receive certain combination drug therapies.

Pancreatic adenocarcinoma remains one of the deadliest forms of oncology in the U.S., accounting for up to 95% of the roughly 67,000 annual pancreatic cancer cases identified by the National Cancer Institute.

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While the disease makes up only 3.2% of total cancer diagnoses, it causes a disproportionately high number of cancer deaths because it is often found late and can spread quickly.

The new drug was approved after a randomized, open-label trial of 500 patients with pre-treated metastatic disease. Patients taking Rasonque achieved a median overall survival of 13.2 months, nearly double the 6.7-month survival rate seen in the standard chemotherapy control group.

"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.

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Regulators accelerated the medication's path to market by granting it priority review, orphan drug status and a breakthrough therapy designation.

Additionally, the filing was evaluated using the FDA commissioner’s National Priority Voucher pilot program to speed up access to critical public health treatments.

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

Safety profiles show that the most frequent adverse events linked to Rasonque included rash, diarrhea, mouth sores (stomatitis), nausea, fatigue, vomiting, abdominal pain, fluid retention (edema), reduced appetite and bleeding (hemorrhage).

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"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."