FDA Approves New Pancreatic Cancer Treatment Pill

The U.S. Food and Drug Administration (FDA) has approved a new cancer treatment pill named Rasonque, manufactured by Revolution Medicines based in California. This medication serves as an inhibitor for the most prevalent type of pancreatic cancer and is taken once daily. It targets various forms of a protein known as RAS, which promotes tumor growth.

A statement from the FDA highlighted Rasonque as a ‘new treatment option’ for patients with advanced pancreatic cancer. The agency approved the drug significantly ahead of its review deadline by more than six months.

‘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,’ stated acting FDA Commissioner Kyle Diamantas. He expressed pride in FDA scientists for their fast and thorough review process and unwavering commitment that led to this milestone.

Rasonque is designed for adult patients with metastatic pancreatic cancer who have either not responded to at least one prior treatment or are ineligible for certain combination drug therapies. Pancreatic adenocarcinoma is one of the deadliest cancer forms in the U.S., accounting for up to 95% of the approximately 67,000 annual pancreatic cancer cases, according to the National Cancer Institute.

The disease represents only 3.2% of total cancer diagnoses but causes a disproportionately high number of cancer deaths due to its late detection and rapid spread. The medication received approval following a randomized, open-label trial involving 500 patients with pre-treated metastatic disease. Those treated with Rasonque had a median overall survival of 13.2 months, nearly double the 6.7-month survival rate of patients receiving standard chemotherapy.

‘This drug showed unprecedented results in an area of high unmet need,’ remarked Dr. Angelo de Claro, director of the FDA’s Oncology Center of Excellence.

The drug’s expedited approval process was facilitated by several designations granted by regulators, including priority review, orphan drug status, and breakthrough therapy designation. The FDA commissioner’s National Priority Voucher pilot program also played a role in accelerating access to this critical treatment.

Ahead of official approval, a ‘safe to proceed’ letter issued by the agency in May enabled an expanded access protocol, allowing patients access to the drug. Common side effects noted during clinical trials 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,’ commented Dr. Anna Berkenblit, chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN). She believes the drug will transform pancreatic cancer treatment, offering patients more time with loved ones and possibly improving their quality of life.