The U.S. Food and Drug Administration has approved Rasonque, an experimental once-daily pill for adults with advanced pancreatic cancer, months ahead of its scheduled review date. Manufactured by Revolution Medicines, the drug targets multiple forms of the RAS protein, which is a primary driver of tumor growth in the most common type of pancreatic cancer.
The approval is for patients with metastatic pancreatic adenocarcinoma whose disease has not responded to at least one prior treatment or who are not candidates for certain combination drug therapies. Pancreatic adenocarcinoma is one of the deadliest cancers in the U.S., accounting for a significant portion of cancer deaths despite making up a smaller percentage of total cancer diagnoses.
In a randomized, open-label trial involving 500 patients with pre-treated metastatic disease, those taking Rasonque achieved a median overall survival of 13.2 months. This is nearly double the 6.7-month survival rate observed in the standard chemotherapy control group. The FDA granted Rasonque Priority Review, Orphan Drug status, and a Breakthrough Therapy designation, accelerating its path to market.
Acting FDA Commissioner Kyle Diamantas stated that the approval provides a critical new option for patients facing a historically hard-to-treat cancer. Anna Berkenblit, M.D., chief scientific and medical officer of the Pancreatic Cancer Action Network, called the approval a landmark advance that could transform treatment and offer patients more time and optimism.
Common side effects reported during clinical trials included rash, diarrhea, stomatitis, nausea, fatigue, vomiting, abdominal pain, edema, reduced appetite, and hemorrhage.