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FDA approves once-daily pill for pancreatic cancer after trial shows survival nearly doubles
The FDA approved Rasonque, a once-daily pill for advanced pancreatic cancer, after a trial showed patients taking it survived a median of 13.2 months versus 6.7 months on standard chemotherapy. The drug targets RAS proteins and was granted priority review and breakthrough therapy status.
The U.S. Food and Drug Administration on Wednesday approved a new once-daily pill for advanced pancreatic cancer, a treatment that nearly doubled survival rates in a clinical trial. The drug, Rasonque, is made by Revolution Medicines and is designed to inhibit multiple forms of the RAS protein, a key driver of tumor growth in the most common type of pancreatic cancer.
The approval applies to adults with metastatic pancreatic adenocarcinoma whose disease did not respond to at least one prior treatment or who could not receive certain combination drug therapies. In a press release, the FDA called Rasonque a “new treatment option for patients with advanced pancreatic cancer” and noted that the agency cleared it more than six months ahead of its review deadline.
“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. “I am immensely proud of the dedicated FDA scientists whose fast, thorough review and relentless commitment made this groundbreaking milestone a reality.”
The authorization was based on a randomized, open-label trial involving 500 patients with pre-treated metastatic disease. Patients taking Rasonque achieved a median overall survival of 13.2 months, nearly double the 6.7 months seen in the standard chemotherapy control group. “This drug showed unprecedented results in an area of high unmet need,” said Dr. Angelo de Claro, director of the FDA’s Oncology Center of Excellence.
Pancreatic adenocarcinoma is one of the deadliest forms of cancer in the United States, accounting for up to 95% of the roughly 67,000 annual pancreatic cancer cases identified by the National Cancer Institute. Although the disease represents only about 3.2% of all cancer diagnoses, it causes a disproportionately high number of cancer deaths because it is often detected late and can spread quickly.
The FDA accelerated the drug’s path to market by granting it priority review, orphan drug status, and a breakthrough therapy designation. The filing was also evaluated under the FDA commissioner’s National Priority Voucher pilot program, which is designed to speed access to critical public health treatments. Ahead of the official approval, 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 data from the trial showed that the most frequent adverse events linked to Rasonque included rash, diarrhea, mouth sores, nausea, fatigue, vomiting, abdominal pain, fluid retention, reduced appetite, and bleeding.
“Today’s landmark approval is the most significant advance we have seen in the fight against pancreatic cancer, a devastating disease,” said Dr. Anna Berkenblit, chief scientific and medical officer of the Pancreatic Cancer Action Network. “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.”
