It’s been a long time coming, but a new frontline therapy for pancreatic cancer has been approved by the U.S. Food and Drug Administration (FDA).
While decades of cancer research have led to innovation and new medications for myriad types of cancer, new therapies have yet to be offered for pancreatic cancer until now, giving patients who suffer from it more treatment options.
NALIRIFOX, produced by Ipsen Biopharmaceuticals, is meant for treating metastatic pancreatic adenocarcinoma. It went through the FDA’s orphan drug status approval process and received approval on Tuesday. An orphan drug is intended for a rarer disease, and the program incentivizes drugmakers to produce it to make up for reduced profits.
NALIRIFOX is administered intravenously for 90 minutes every two weeks as part of a rigorous chemotherapy regimen.
A Silent Killer
Pancreatic cancer is one of the most difficult to treat and is the third leading cause of cancer-related death, behind lung and colorectal cancer in the United States. Globally, it is the seventh leading cause of cancer death. According to the Pancreatic Cancer Action Network (PCAN), it’s on track to becoming the second leading cause.
Metastatic pancreatic adenocarcinoma starts in the pancreas but often spreads to other organs, such as the abdomen, liver, lungs, bones, and brain. It is not often that patients diagnosed with metastatic pancreatic adenocarcinoma live longer than five years; according to Johns Hopkins Medicine, the five-year survival rate is just 5 percent to 10 percent.
The survival rate is so low because symptoms of pancreatic cancer can be challenging to pinpoint. They are often indistinct and imitate symptoms of everyday illnesses, such as belly pain, loss of appetite, itching, and bowel issues. Because of this, pancreatic cancer is often diagnosed in its later stages, making for difficult prognoses and low survival rates. Currently, no standard screening tests exist to detect pancreatic cancer early.
But researchers and physicians alike are hopeful that NALIRIFOX, a combination of irinotecan liposome, oxaliplatin, fluorouracil, and leucovorin, will be the new first-line treatment for this cancer.
“We are pleased that the FDA has approved the NALIRIFOX regimen. For a tough disease with few treatment options, this is significant news for people with metastatic pancreatic ductal adenocarcinoma (PDAC) given it has been more than 10 years since there has been a PDAC-specific approval in the first-line setting,” Julie Fleshman, president and CEO of the PCAN, said in a press statement.
Increased Survival Rate, Muted Cancer Progression
The medication received its FDA approval after undergoing an open-label, active-controlled trial that included 770 patients who all had metastatic pancreatic adenocarcinoma. None of the patients had received traditional chemotherapy treatment. Patients were randomly divided into groups to receive different therapy regimens, including standard-of-care chemotherapy treatments plus NALIRIFOX or just chemotherapy.
The trial found that patients who underwent therapy that included NALIRIFOX had a median overall survival rate of 11.1 months, while those who underwent standard chemotherapy had a median overall survival rate of 9.2 months. The study also found that cancer progression was muted in patients who received treatment with irinotecan liposome; the median progression-free survival rate was 7.4 months versus 5.6 months for patients who received traditional chemotherapy.
Common side effects of NALIRIFOX during the study included diarrhea, fatigue, nausea, vomiting, reduced appetite, abdominal pain, mucosal inflammation, constipation, and weight loss. Other side effects included lab abnormalities, such as decreased blood cell counts and potassium.
“We thank Ipsen for seeking FDA approval for this regimen and are thankful for the patients who participated in the 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,” said Ms. Fleshman.
