FDA approval opens new chapter in pancreatic cancer treatment

by University of Miami Leonard M. Miller School of Medicine

edited by Sadie Harley, reviewed by Andrew Zinin

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For decades, a diagnosis of metastatic pancreatic cancer left patients and physicians facing the same harsh reality: limited options, difficult treatments and too little time. Now, a pill tested in a global clinical trial may begin to change that.

The oral targeted therapy helped patients live nearly twice as long as those who received standard chemotherapy, marking a major advance for one of cancer's most difficult diseases. Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, participated in the global study and was the only South Florida site to offer the trial.

The U.S. Food and Drug Administration has approved Rasonque, the brand name for daraxonrasib, a first-in-class targeted therapy for adults with metastatic pancreatic adenocarcinoma. The approval was based on the Phase III RASolute 302 trial, which enrolled 500 patients across 59 sites in six countries.

Patients who took the pill lived a median of 13.2 months, nearly twice as long as those who received standard chemotherapy.

A milestone years in the making

"This is a monumental shift in the treatment of pancreatic cancer," said Dr. Peter Hosein, co-leader of Sylvester's Gastrointestinal Cancer Site Disease Group. "It's a historic day for pancreatic cancer."

The approval was broader than investigators expected. The trial required KRAS mutation testing, but the FDA approved the therapy for eligible patients regardless of KRAS mutation status.

"For a drug that has the potential to benefit a broad range of patients with metastatic pancreatic cancer, this is unprecedented," Hosein said.

"This approval is a landmark for patients, but it is also an important scientific milestone because it demonstrates that RAS, one of the most important drivers of pancreatic cancer, can be successfully targeted," said Dr. Nipun Merchant, founding director of the Sylvester Pancreatic Cancer Research Institute (SPCRI) and the Alan Livingstone Professor in the DeWitt Daughtry Family Department of Surgery.

"At the Sylvester Pancreatic Cancer Research Institute, our goal is to build on advances like this by bringing together clinical trials, patient-derived biospecimens and laboratory investigation to understand why some tumors respond, why resistance develops and how we can make these responses deeper and more durable."

More than a new treatment

For Hosein, the approval offers patients something less easily captured in clinical data: hope. "A lot of times when people come in with a new diagnosis of pancreatic cancer, they are depressed and their whole world is turned upside down," he said. "Knowing that there's something like this, which is such a big breakthrough, which is also not chemotherapy, is a big deal."

Sylvester's role grew from its participation in multicenter clinical trials and pancreatic cancer research programs. The cancer center became the first Florida site to open an Expanded Access Program for daraxonrasib, allowing eligible patients to receive the therapy before approval. More than 80 patients enrolled in approximately three months.

Hosein has also helped design RASolute 304, a new study testing whether daraxonrasib can prevent pancreatic cancer from returning after surgery.

The next scientific question: Resistance

A biospecimen initiative is collecting blood and tumor samples to study why some tumors evade RAS inhibition. RAS is a family of proteins that drives the growth of most pancreatic cancers and has long been one of the most difficult targets in cancer biology.

That effort is closely integrated with laboratory research at the SPCRI aimed at defining the mechanisms that allow pancreatic cancers to escape RAS inhibition.

Merchant; Dr. Jashodeep Datta, the DiMare Family Endowed Chair in Immunotherapy and co-leader of the Gastrointestinal Cancer Site Disease Group; and Nagaraj Nagathihalli, Ph.D., associate professor in the DeWitt Daughtry Family Department of Surgery and assistant director of basic science research at the SPCRI, are working collaboratively to understand how tumor-intrinsic changes and interactions within the tumor microenvironment contribute to treatment resistance and identify strategies to prevent or overcome it.

"FDA approval is an extraordinary milestone, but it is also the beginning of the next set of scientific questions," Datta said.

"The challenge now is understanding why some tumors eventually evade RAS inhibition and how we can develop new strategies to prevent or overcome that resistance. Through ongoing collaborations with Revolution Medicines and other industry partners, we are laser-focused on understanding and targeting factors in the tumor microenvironment that provoke and sustain resistance to RAS inhibition in pancreatic cancer."

What comes next

Sylvester is enrolling patients in the RASolute 304 study, a Phase III trial testing whether daraxonrasib can reduce recurrence after pancreatic cancer surgery.

Researchers also opened a study for newly diagnosed patients with metastatic pancreatic cancer that combines chemotherapy with a KRAS-targeted therapy.

"With other ongoing trials, it is conceivable that in the future, we will recommend this pill for someone with newly diagnosed pancreatic cancer rather than chemotherapy infusions, which have been the standard for decades," Hosein said.

It also shows how today's breakthrough can seed tomorrow's research. Research underway across SPCRI is already focused on the next phase: understanding why tumors adapt, identifying mechanisms of resistance and developing strategies to overcome them, and building on the progress now reaching patients.

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Oncology Provided by University of Miami Leonard M. Miller School of Medicine Who's behind this story?

Sadie Harley

BSc Life Sciences & Ecology. Microbiology lab background with pharmaceutical news experience in oil, gas, and renewable industries. Full profile →

Andrew Zinin

Master's in physics with research experience. Long-time science news enthusiast. Plays key role in Science X's editorial success. Full profile →

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