Ipsen's Onivyde regimen, now FDA-approved as first-line therapy for metastatic pancreatic adenocarcinoma, offers hope for a disease with dismal survival rates. The approval follows successful trials and recognition in treatment guidelines.

France: On February 13, 2024, Ipsen announced the FDA's approval of the supplemental new drug application for Onivyde (irinotecan liposome injection) in combination with oxaliplatin, fluorouracil, and leucovorin (NALIRIFOX) as a first-line therapy for adults with metastatic pancreatic adenocarcinoma (mPDAC). This approval marks the second endorsement for an Onivyde regimen in mPDAC, following the FDA's 2015 approval of Onivyde combined with fluorouracil and leucovorin after disease progression with gemcitabine-based therapy.

Pancreatic adenocarcinoma (PDAC) is prevalent, with over 60,000 cases diagnosed annually in the U.S. and nearly 500,000 globally. Early detection is challenging due to vague symptoms, leading to late-stage diagnoses, and a grim prognosis with less than 20% survival beyond one year. Pancreatic cancer holds the lowest five-year survival rate globally and, in the U.S.

The FDA's approval was grounded in efficacy and safety data from the NAPOLI 3 trial, a Phase III study involving 770 untreated adults with mPDAC across 18 countries. This study, meeting primary and secondary endpoints, was presented at the ASCO Gastrointestinal conference 2023, and published in The Lancet. Additionally, NALIRIFOX earned recognition in the National Comprehensive Cancer Network (NCCN) guidelines as a preferred Category 1 treatment for first-line metastatic disease and a preferred option for first line locally advanced disease.

Onivyde functions by inhibiting topoisomerase I, an enzyme crucial for DNA replication in cancer cells, leading to their demise. Encased in liposomes, irinotecan in Onivyde accumulates in tumors, providing sustained release. Administered via intravenous infusion every two weeks, dosing modifications are recommended. Eligible individuals in the U.S. can access Onivyde immediately if treatment-naïve or post-gemcitabine therapy.

In 2020, the FDA granted Onivyde Fast Track designation for its potential as a first-line combination therapy for mPDAC, underscoring its significance in addressing unmet medical needs.

Christelle Huguet, EVP and Head of Research and Development, Ipsen said, “The results from the Phase III NAPOLI 3 trial represent the first positive data for an investigational regimen in first-line metastatic pancreatic adenocarcinoma versus the currently approved nab-paclitaxel and gemcitabine regimen. With today’s approval, this Onivyde (NALIRIFOX) regimen can now offer a potential new standard-of-care treatment option with proven survival benefits for people living with metastatic pancreatic adenocarcinoma in the U.S.”

Dr. Zev Wainberg, Professor of Medicine and Co-Director of the UCLA GI Oncology Program said, “This approval is a testament to our commitment and unwavering support of patients and caregivers to lessen the symptoms of agitation associated with dementia due to Alzheimer’s disease. We look forward to offering this first FDA-approved treatment option to address this significant unmet need for patients. We are grateful to the patients and caregivers who participated in these important trials. The approval of this Onivyde regimen is an important milestone for people living with mPDAC, their families and healthcare providers, with the NAPOLI 3 trial having demonstrated survival benefits versus a current standard of care treatment option.”

Julie Fleshman, JD, MBA, President and CEO of Pancreatic Cancer Action Network (PanCAN) said, “We are pleased that the U.S. Food and Drug Administration has issued this new approval of the NALIRIFOX regimen. 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. We are thankful to the patients who participated in this clinical trial as they play a crucial role in advancing treatments for pancreatic cancer.”

According to TechSci Research, The FDA approval of Ipsen's Onivyde regimen as a first-line therapy for metastatic pancreatic adenocarcinoma is poised to significantly impact the market landscape. With pancreatic cancer representing a substantial medical challenge and having one of the lowest survival rates among cancers, this approval marks a significant advancement in treatment options. As a result, Ipsen is likely to experience a surge in demand for Onivyde, driving revenue growth and potentially solidifying its position as a key player in the oncology market. Additionally, the expanded indication for Onivyde opens new opportunities for Ipsen to capture market share, particularly in the segment of patients who are treatment-naïve or have experienced disease progression following gemcitabine-based therapy. This could translate into competitive advantages over other pharmaceutical companies operating in the oncology space.

Furthermore, the FDA's Fast Track designation for Onivyde underscores its perceived clinical significance and may expedite its adoption by healthcare providers, further boosting its market penetration. Ipsen's patient support program, Ipsen Cares, not only facilitates access to Onivyde but also enhances the company's reputation for patient-centric care, potentially strengthening brand loyalty and market positioning. However, the market response could also be influenced by factors such as pricing, reimbursement, and competition from existing and emerging therapies. Market dynamics, including shifts in treatment guidelines and evolving patient preferences, will also play a role in determining the long-term impact of Onivyde's approval on the market.