The U.S. Food and Drug Administration approved daraxonrasib on Wednesday, marking the first time regulators have cleared an oral targeted pill for pancreatic cancer. Marketed under the brand name Rasonque by Revolution Medicines, the once-daily tablet achieved a median overall survival of 13.2 months in a 500-patient clinical trial, nearly doubling the 6.7-month survival rate seen with standard chemotherapy.
Clinical Trial Results and Survival Rates
Pancreatic adenocarcinoma accounts for up to 95 percent of the approximately 67,000 annual pancreatic cancer cases identified by the National Cancer Institute. The randomized, open-label trial evaluated 500 adult patients with metastatic disease that had either failed at least one prior treatment or proved ineligible for certain combination drug therapies. Patients who received daraxonrasib reached a median overall survival of 13.2 months, compared to 6.7 months for those in the standard chemotherapy control group.
Dr. Angelo de Claro, director of the FDA’s Oncology Center of Excellence, stated that the drug demonstrated unprecedented results in an area of high unmet medical need. Regulators accelerated the review process by granting the medication priority review, orphan drug status, and a breakthrough therapy designation, ultimately approving the tablet more than six months ahead of its scheduled review deadline.
Targeting the ‘Undruggable’ RAS Protein
Mutations in the RAS gene family drive tumor growth in roughly 90 percent of all pancreatic cancers. For decades, the structural properties of these mutated proteins prevented medications from binding to them, earning the RAS family a reputation among scientists as an undruggable target. Rasonque functions as an inhibitor that directly blocks multiple forms of the overactive RAS protein to halt cancer cell proliferation.
Acting FDA Commissioner Kyle Diamantas noted that the authorization provides a critical new option for patients facing a historically difficult-to-treat cancer.
Safety Profile and Side Effects
Clinical trials tracked several adverse events associated with daily administration of the tablet. The most frequent side effects reported by patients included skin rash, diarrhea, mouth sores or stomatitis, nausea, fatigue, vomiting, abdominal pain, fluid retention or edema, reduced appetite, and bleeding or hemorrhage. Regulatory safety reviews established that these side effects occurred while patients experienced improved overall survival compared to traditional chemotherapy regimens.
Patient Impact and Outlook
The approval offers a new treatment avenue for individuals diagnosed late in the progression of the disease. Dr. Céline Gounder, a medical correspondent, pointed out that the majority of patients receive their diagnosis after the cancer has already metastasized. Oncology specialists across the country, including Dr. Pashtoon Kasi of City of Hope Orange County and Dr. Andrew Coveler of the Fred Hutch Cancer Center, view the authorization as a major step forward that could pave the way for similar targeted therapies in other malignancies.
Keep reading