For Advanced Pancreatic Cancer Patients Running Out of Treatments, a New Pathway Offers Renewed Hope Thanks to the FDA Granting ‘Compassionate Use’ Access to Daraxonrasib

SurvivorNet is breaking down what this decision means for patients navigating their treatment options.

In early May, the U.S. Food and Drug Administration (FDA) issued a “safe to proceed” letter to Revolution Medicines, authorizing the drug company to launch an Expanded Access Program (EAP) for daraxonrasib.

“We anticipate the daraxonrasib will move up into earlier lines of therapy and not just daraxonrasib, but RAS inhibitors in general,” Dr. Anna Berkenblit, Chief Scientific and Medical Officer at Pancreatic Cancer Action Network (PanCAN), tells SurvivorNet.

“If it works this well in patients with previously treated metastatic disease, we’re really excited about the potential of it working even better in frontline or untreated metastatic patients, and maybe even in the adjuvant [after surgery] setting for the small percent of patients who are diagnosed with early-stage, localized disease that can be surgically removed.”

Under FDA rules, expanded access allows larger groups of patients to receive investigational medicines under a monitored treatment protocol.

The goal is treatment—not research—and the program must follow strict safety and reporting requirements.

Drug companies can sometimes help patients get into clinical trials, and in certain situations, they may also offer what’s called ‘compassionate use’ for patients who are seriously ill and trying a new drug that hasn’t been fully approved yet is a viable option. These special cases apply to patients, usually only after all standard treatments have stopped working.

Patients or doctors may also look into whether a drug can be used ‘off‑label,’ which means using an FDA‑approved drug for a different condition than the one it was originally approved to treat.

The Food and Drug Administration offers a Compassionate Use Resources Page for patients seeking additional ways to access this program.

What “Expanded Access” Means

The FDA’s expanded access or “compassionate use” program allows patients, through their licensed physicians, to request investigational treatments when no other viable options remain.

Both the FDA and the drug manufacturer must approve each request.

Patients receiving a drug through expanded access are monitored with the same scrutiny seen in clinical trials.

WATCH: Dr. Neel spoke with SurvivorNet about why he would choose a clinical trial for himself.

“Compassionate Use” can also manifest in other ways, particularly when the patient meets the criteria for the treatment but lacks the financial means to obtain it. This is something Revolution Medicines offers, as we’ll explain further shortly. Again, as long as your doctor can demonstrate that you are receiving continued clinical benefit from the drug.

Revolution Medicines’ Expanded Access Criteria

Revolution Medicines’ daraxonrasib EAP is available to U.S. patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC) who:

• Are not eligible for a Revolution Medicines–sponsored clinical trial or cannot access one
• Have no satisfactory alternative treatment options

Revolution Medicines says the investigational drug is provided at no cost, though the company does not reimburse patients for time or participation.

All requests must be submitted by a licensed treating physician; the company cannot accept inquiries directly from patients or caregivers.

Physicians can initiate requests or ask questions by contacting [email protected].

WATCH: Why Pancreatic Cancer Is Difficult to Treat

Daraxonrasib is designed to target the KRAS‑driven biology behind most pancreatic tumors.

KRAS mutations appear in roughly 90% of pancreatic cancer cases, yet until now, no therapy has effectively exploited this vulnerability.

In the Phase 3 RASolute‑302 trial, the drug more than doubled median overall survival—13.2 months compared with 6.7 months on standard chemotherapy—offering new hope for patients who have historically had few effective options.

Following the FDA’s authorization, Revolution Medicines said in a statement it is “moving as quickly as possible to ensure safe and equitable access to daraxonrasib for eligible patients in the United States.”

The company emphasized that expanded access is not a substitute for clinical trial participation and does not generate research data.

RELATED: What Does it Mean to Try an “Off-label” or “Experimental” Cancer Treatment?

Why Daraxonrasib Helps Meet an Unmet Need So Desperately?

Pancreatic cancer is notoriously difficult to detect early, often remaining silent until it has advanced. Its biology also makes it challenging to treat.

“Around eighty percent of pancreatic cancer patients already have advanced disease by the time they’re diagnosed, severely limiting treatment options,” explains Dr. Anirban Maitra, Co-Leader of the Pancreatic Cancer Moon Shot at MD Anderson Cancer Center.

“Just twenty percent of patients have their cancer caught early enough to make them a candidate for surgery, the only way pancreatic cancer can be cured.”

Dr. Allyson Ocean of Weill Cornell explains that pancreatic tumors are surrounded by a dense, protective stroma that blocks chemotherapy and radiation from reaching cancer cells effectively. However, new developments, like daraxonrasib, are shifting what’s possible.

WATCH: A Potential New Era Of Targeted Therapy For Pancreatic Cancer

A drug called daraxonrasib is being hailed as potentially “game‑changing” for patients with Kirsten rat sarcoma viral oncogene homolog (KRAS)‑mutated pancreatic cancer — a gene mutation present in roughly 90% of cases and historically considered nearly untreatable.

KRAS has long been a difficult target for treatment because its smooth structure lacks the pockets where most drugs typically bind to become effective.

“It’s a very smooth protein. It doesn’t have places for drugs to grab onto,” Dr. Nicholas Hornstein of Northwell’s Lenox Hill Hospital told SurvivorNet.

Daraxonrasib works differently. Acting as a kind of “molecular glue,” it binds the KRAS gene to another protein, locking it in an inactive state. This mechanism may allow it to work across multiple KRAS mutations, potentially benefiting a wider group of patients who carry the KRAS gene.

The Phase 3 RASolute 302 clinical trial tested daraxonrasib against standard chemotherapy in patients with previously treated metastatic pancreatic cancer.

For clinicians, the implications are significant.

“If I have an 81‑year‑old who doesn’t look like a candidate for chemotherapy, now I have an option that isn’t chemo,” Dr. Hornstein said.

Who Can Benefit From Daraxonrasib?

The patients included in the phase 3 study had metastatic PDAC that was refractory (non-responsive) to prior treatment, but Dr. Berkenblit predicts that the drug may have uses in earlier-stage disease as well.

“If the FDA approves the drug after reviewing the efficacy and safety data, the initial population would be patients with previously treated metastatic pancreatic cancer. That’s just the beginning,” Dr. Berkenblit explains.

Initially, patients would need to meet certain requirements to get daraxonrasib, including:

• Measurable metastatic disease
• An acceptable performance status allows them to conduct most daily activities
• Well-functioning organs

Again, in the trial, Daraxonrasib was compared to the standard chemotherapy.

Questions to Ask Your Doctor

If you are facing a pancreatic cancer diagnosis, you may have questions but are unsure how to get the answers you need. SurvivorNet suggests asking your doctor the following to kickstart your journey to more solid answers.

• What type of pancreatic cancer do I have?
• Has my cancer spread beyond my pancreas?
• If so, where has it spread, and what is the stage of the disease?
• What is my prognosis?
• What are my treatment options?
• What side effects should I expect after undergoing treatment?
• Will insurance cover my recommended treatment?

Learn more about SurvivorNet's rigorous medical review process.

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