FDA Approves New First-Line Treatment Option for Metastatic Pancreatic Cancer: What You Need to Know

For the first time in more than a decade, the FDA has approved a new first-line treatment for patients with metastatic pancreatic cancer. After a clinical trial showed a positive survival benefit, the combination chemotherapy called NALIRIFOX is now approved for patients who have not received any previous treatment. For a disease with limited treatment options, today’s FDA announcement is exciting news.

“With each new approved treatment, there is more hope for those who will be diagnosed in the future and people currently living with pancreatic cancer may have more time with their loved ones,” said PanCAN President and CEO Julie Fleshman, JD, MBA. “We are also thankful for the patients who participated in this clinical trial. The only way to bring new treatment options to patients is through lab-based research and clinical trials. PanCAN remains committed to providing evidence-based information and resources to patients and caregivers and advancing research to improve patient outcomes.”

Here, PanCAN answers questions related to this new treatment option.

What is NALIRIFOX?

NALIRIFOX is a chemotherapy treatment. It is a combination of three previously approved pancreatic cancer drugs, liposomal irinotecan (Nal-IRI or Onivyde®), made by the pharmaceutical company Ipsen, plus 5 fluorouracil (5-FU)/leucovorin and oxaliplatin. NALIRIFOX will be delivered intravenously (IV, through a vein under the skin).

What does this FDA approval mean?

NALIRIFOX has been approved by the FDA as a new first-line treatment for metastatic pancreatic cancer. This means patients whose cancer has spread and who have not had treatment yet can now receive this drug combination.

Are these new drugs?

No. All of the drugs in NALIRIFOX have already been approved for other purposes; what is new is the combination of these drugs together as a first-line treatment.

Liposomal irinotecan, in combination with 5-FU/leucovorin, is already approved for people with metastatic pancreatic cancer that has continued to grow after being treated with another chemotherapy called gemcitabine (Gemzar®). Oxaliplatin has also been approved and used to treat other cancers for a long time.

NALIRIFOX is a combination of liposomal irinotecan, 5-FU/leucovorin and oxaliplatin. This combination has now been approved for a new group of patients, those with metastatic pancreatic cancer who have not had any other treatment. This is the first approval for a first-line treatment for metastatic pancreatic cancer in over ten years.

What is the survival benefit?

In a clinical trial, the NALIRIFOX regimen was compared to gemcitabine (Gemzar) plus nab-paclitaxel (Abraxane®), which is one of the current standard-of-care treatments for patients with metastatic pancreatic cancer. The results, published in October 2023, showed that patients treated with NALIRIFOX had an overall survival of 11.1 months, which was a statistically significant improvement over the 9.2-month overall survival with gemcitabine and nab-paclitaxel.

What are the side effects?

In the clinical trial, patients took NALIRIFOX for a median of six weeks longer than those receiving gemcitabine and nab-paclitaxel, showing that NALIRIFOX was relatively well tolerated. The most frequent side effects reported in the NALIRIFOX group included neutropenia (low levels of a type of immune cell called neutrophils) and hypokalemia (low potassium level), and gastrointestinal disorders like diarrhea and nausea.

Is NALIRIFOX the same as FOLFIRINOX?

The combination chemotherapy FOLFIRINOX is composed of 5-FU, leucovorin, irinotecan and oxaliplatin. In 2010, a clinical trial showed that FOLFIRINOX was effective for the treatment of metastatic pancreatic cancer in people who hadn’t received prior treatment.

The drug liposomal irinotecan replaces irinotecan to make NALIRIFOX. Liposomal irinotecan is a modified form of irinotecan, designed to stay in the body longer before it gets broken down.

Does insurance cover this treatment?

FDA approval means this drug combination is safe and effective, and although the FDA does not decide what is covered by insurance, when a drug gets FDA approval Medicare and Medicaid will usually cover it.  Coverage for chemotherapy drugs will vary based on the specific plan and insurance company a person uses.

Contact PanCAN Patient Services for more information on financial assistance programs for those experiencing or anticipating cost-related barriers to care.

I am a patient with pancreatic cancer interested in NALIRIFOX. What should I do?

People diagnosed with pancreatic cancer should talk to their healthcare team about this treatment option. Since this approval is for first-line treatment (the first or initial treatment a person receives after diagnosis), it will impact people who have not received treatment for their pancreatic cancer yet.

For people who have already received treatment with a drug called gemcitabine, a similar chemotherapy containing one of the drugs in NALIRIFOX, liposomal irinotecan, is also approved.

Contact PanCAN Patient Services for additional information and support, including information on what questions to ask and how to seek out a second opinion. Our Case Managers can help you understand your options and connect you with resources to learn more.