The Food and Drug Administration has approved Rasonque, a new targeted treatment for certain adults with metastatic pancreatic adenocarcinoma. A clinical trial found that the drug nearly doubled median overall survival compared with standard chemotherapy.
Rasonque, also known as daraxonrasib, is a once-daily tablet developed by Revolution Medicines. It targets multiple forms of RAS, a protein that drives tumor growth in most patients with pancreatic adenocarcinoma.
The FDA approved the drug for adults with metastatic pancreatic adenocarcinoma who have received at least one previous systemic therapy. It is also approved for patients who are not candidates for multiagent systemic therapy, which uses two or more treatments.
The decision was based on a randomized, open-label clinical trial involving 500 adults with previously treated metastatic pancreatic adenocarcinoma. Patients who received Rasonque had a median overall survival of 13.2 months. The median was 6.7 months among those treated with standard chemotherapy.
“Today’s approval provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer,” Acting FDA Commissioner Kyle Diamantas said.
Pancreatic adenocarcinoma begins in cells lining the ducts of the pancreas. It accounts for approximately 90 to 95 percent of the roughly 67,000 new pancreatic cancer cases diagnosed in the United States each year, according to the National Cancer Institute.
Although the disease represents about 3.2 percent of all cancer diagnoses, it accounts for a disproportionately large share of cancer deaths. The FDA attributed that high death toll to late detection, the disease’s aggressive course, and historically limited treatment options.
Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research at the Dana-Farber Cancer Institute, called the approval an “exceptional moment” for pancreatic cancer patients.
“It brings a new treatment option and new hope to patients with metastatic pancreatic cancer, while giving us a foundation to make many more advances against this difficult disease,” Wolpin said in a statement to ABC News.
The FDA previously granted Rasonque Breakthrough Therapy and Orphan Drug designations. The drug also received Priority Review.
Its application was reviewed through the Commissioner’s National Priority Voucher pilot program. The program is intended to accelerate reviews of treatments that address national public health priorities.
The agency approved Rasonque 6.5 months before its user fee deadline.
“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.
Before the approval, the FDA issued a “safe to proceed” letter in May that allowed Revolution Medicines to begin an expanded-access treatment protocol. The decision enabled eligible patients to receive the investigational drug under FDA regulations while its application remained under review.
The most common side effects of Rasonque include rash, diarrhea, inflammation of the mouth’s mucous membranes, nausea, fatigue, vomiting, abdominal pain, swelling, decreased appetite, and bleeding.