Oncologist Explains What the Rasonque Approval Means for Patients With Metastatic Pancreatic Cancer

The U.S. Food and Drug Administration’s approval of Rasonque (daraxonrasib) represents an important advance for patients with metastatic pancreatic cancer. Dr. Kristin Higgins, radiation oncologist and chief clinical officer for City of Hope Atlanta, was involved in the phase 3 clinical trial that helped lead to the approval.

Higgins spoke with CURE about what the approval means for patients, how Rasonque works, the clinical trial results, potential side effects and what the treatment could mean for the future of pancreatic cancer care.

Cure: What Does the Rasonque Approval Mean for Patients With Pancreatic Cancer?

Dr. Kristin Higgins: Patients with pancreatic cancer have had very limited treatment options and survival for many years. There have been few new drug approvals for this disease, which has contributed to poor long-term survival.

Rasonque represents one of the first major advances in pancreatic cancer treatment in decades. Results from the phase 3 trial showed that patients who received the treatment after standard chemotherapy had their survival nearly doubled compared with standard chemotherapy.

That is a significant benefit, especially for a patient population that has historically had relatively few treatment advances.

The approval is also significant because Rasonque is the first targeted therapy approved for pancreatic cancer. Targeted therapies work by focusing on specific molecular pathways involved in cancer growth. We have seen this approach make a major difference in cancers such as lung cancer, but pancreatic cancer has not had an approved targeted therapy until now.

For patients, that means there is more hope and another treatment option after standard chemotherapy.

How Does Rasonque Work to Treat Pancreatic Cancer?

Rasonque is a pan-RAS inhibitor. It works by blocking signaling pathways downstream of RAS that help cancer cells grow.

This is different from traditional chemotherapy, which can affect cancerous and healthy cells throughout the body and lead to more generalized side effects.

Targeted therapies are designed to be more specific to the biology of the cancer. In general, they can be better tolerated and may allow patients to maintain a better quality of life compared with traditional chemotherapy.

What Did the Rasonque Clinical Trial Show About Survival?

The results are important because they show that patients whose cancer progresses after standard chemotherapy still have treatment options.

In the trial, median overall survival was 13.2 months with Rasonque compared with 6.7 months with standard chemotherapy.

For patients, those additional months can mean meaningful time with the people they love and the opportunity to experience important milestones — whether that's attending a daughter's wedding or meeting a new grandchild.

That's what this treatment brings to the table: more time and, importantly, hope.

Who Is Rasonque Approved For?

Currently, Rasonque is approved for patients whose metastatic pancreatic cancer has progressed after frontline chemotherapy.

That means someone who has just been diagnosed with pancreatic cancer would not typically receive Rasonque as their initial treatment. They would first receive standard chemotherapy.

Researchers are now studying whether Rasonque could be beneficial earlier in treatment. Those clinical trials are ongoing, so we do not yet have enough information to know whether the treatment should be used at the time of diagnosis.

For now, patients with newly diagnosed pancreatic cancer would receive standard chemotherapy. If their cancer progresses, Rasonque may then be an option.

What Side Effects Can Patients Experience With Rasonque?

Rasonque is taken once daily. Because it is an oral treatment, the dose can be This advance was made possible because patients participated in clinical trials. Clinical trials are essential for developing new cancer treatments and improving outcomes.

At City of Hope, we participated in the phase 3 trial that led to the FDA approval. We also opened an expanded access program that allowed some patients to receive Rasonque while the treatment was moving through the approval process.

Expanded access programs can be particularly important when promising treatments become available but patients cannot wait several months for an FDA decision.

I also want patients to understand that clinical trials are not necessarily something that only benefits future patients. Patients participating in trials can benefit from access to promising treatments, and in many cases, some of the best cancer care is available through a clinical trial.

What Does the Rasonque Approval Mean for the Future of Pancreatic Cancer Treatment?

I think this is the beginning of precision medicine in pancreatic cancer.

I expect we will see additional targeted therapies approved for pancreatic cancer. Researchers will also learn more about why cancers become resistant to these treatments and how we can overcome that resistance.

Rasonque is an important first step, but there is still a lot to learn. The hope is that this progress will lead to even better outcomes for patients with pancreatic cancer.

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