FDA Approves Breakthrough Pancreatic Cancer Pill That Nearly Doubles Survival Time In Studies
The U.S. Food and Drug Administration has approved daraxonrasib1, sold under name Rasonque, the first targeted therapy of its kind for metastatic pancreatic cancer. This approval comes months ahead of schedule, following a 500-person clinical trial where the once-daily pill nearly doubled median survival compared to standard chemotherapy. Pancreatic cancer has long been considered one of the hardest forms of the disease to treat, making these results even more noteworthy.
What is daraxonrasib & who can take it
Daraxonrasib is a tablet taken once daily that targets multiple forms of a protein called RAS, a key driver of tumor growth in most patients with pancreatic adenocarcinoma. This type of cancer arises from cells lining the ducts of the pancreas, and it's the most common form of pancreatic cancer by far.
In a randomized, open-label, multicenter trial of 500 adults with previously treated metastatic pancreatic adenocarcinoma, daraxonrasib extended survival by nearly double: from an average of 6.7 months with standard chemotherapy to 13.2 months on daraxonrasib.
The study focused on adult with pancreatic adenocarcinoma who have already tried at least one systemic drug treatment, and people who can't take the standard combination of systemic drugs. In other words, this is a new option for patients who've already exhausted, or can't tolerate, existing treatment routes.
Patients interested in the drug should be aware of possible side effects. The most common side effects reported include rash, diarrhea, stomatitis (inflammation of the mouth's mucus membranes), nausea, fatigue, vomiting, abdominal pain, edema, decreased appetite, and hemorrhage. As with any new cancer therapy, patients should talk with their oncology team about what to expect and how side effects will be monitored and managed.
Why pancreatic adenocarcinoma has been so hard to treat
Pancreatic adenocarcinoma accounts for 90% to 95% of the roughly 67,000 new pancreatic cancer cases diagnosed in the U.S. each year, according to the National Cancer Institute. And, despite making up only about 3.2% of all cancer diagnoses, it's responsible for a disproportionately high share of cancer deaths. That's largely because it tends to be caught late, progresses aggressively, and has had frustratingly limited treatment options.
These statistics explain why researchers have been hard at work to find new ways to prevent, diagnose, and treat pancreatic cancer. A new tool from Mayo Clinic could help detect pancreatic cancer earlier than ever, which could make treatments like this one even more effective down the line. There is also a new vaccine showing promise for preventing pancreatic cancer in those at high-risk of the disease.
Fast-tracked approval
Daraxonrasib moved through the FDA on a fast track. Back in May, the FDA issued a "safe to proceed" letter allowing the drug's sponsor to launch an expanded access treatment protocol for daraxonrasib, giving patients access to the investigational drug even before formal approval.
Then, the agency gave daraxonrasib two labels that speed up development and review for drugs treating serious illnesses with few existing options: Breakthrough Therapy and Orphan Drug status. It also got Priority Review, meaning FDA scientists agreed to evaluate the application on a shortened timeline rather than the standard one. On top of that, the application went through a newer FDA pilot program called the Commissioner's National Priority Voucher, which the agency says is meant to speed up reviews of treatments that address major public health needs.
The takeaway
Daraxonrasib's approval marks a shift for a cancer that has historically had few effective treatment options. With ongoing work on earlier detection and even preventive vaccines showing early promise, the outlook for pancreatic cancer patients may be improving on several fronts. If you or someone you love has metastatic pancreatic adenocarcinoma, ask the treating oncologist whether RAS-targeted therapy is an option worth exploring.