Ipsen announces U.S. FDA submission acceptance of its supplemental New Drug Application for Onivyde regimen in first-line metastatic pancreatic ductal adenocarcinoma

• Supplemental New Drug Application (sNDA) submission based on the NAPOLI 3 Phase III trial¹
• Approval would represent expansion of treatment options in an aggressive and difficult-to-treat cancer with few treatment options currently available

PARIS, FRANCE, 14 June 2023 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental new drug application (sNDA) Onivyde (irinotecan liposome injection) plus 5 fluorouracil/leucovorin and oxaliplatin (NALIRIFOX regimen) as a potential first-line treatment for metastatic pancreatic ductal adenocarcinoma (mPDAC). The review is based on positive results from the pivotal Phase III NAPOLI 3 trial, in which the Onivyde regimen demonstrated a statistically significant improvement in overall survival (OS) and progression-free survival (PFS), compared to nab-paclitaxel plus gemcitabine, with a safety profile consistent with the profiles of the treatment components. These results were presented at the January 2023 American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO GI).

“PDAC is a devastating disease in need of additional treatment options. The FDA’s decision to accept the sNDA for this Onivyde-based regimen in treatment-naïve patients with metastatic disease represents an important milestone in the potential treatment of this complex form of cancer,” said Howard Mayer, Executive Vice President and Head of Research and Development at Ipsen. “We’re committed to developing therapies which have the potential to make a meaningful difference to the lives of people living with cancer and look forward to working with FDA as they review this application.”

The FDA provided a Prescription Drug User Fee Act goal date of 13 February 2024 for review of Ipsen’s application. In 2020, the FDA granted Ipsen Fast Track designation for Onivyde as a first-line combination treatment for mPDAC. The FDA’s Fast Track program facilitates the development and expedites the review of medicines that treat serious conditions and have the potential to address an unmet medical need.

NAPOLI 3 data, as presented at ASCO GI 2023

• Trial met its primary endpoint of OS, showing patients in the NALIRIFOX group had a statistically significant improvement in median OS of 11.1 months, versus 9.2 months in the nab-paclitaxel and gemcitabine group (HR 0.83 [95% CI 0.70–0.99]; p=0.04). At 12 months, the OS rate for the NALIRIFOX group was 45.6%, and for the nab-paclitaxel and gemcitabine group it was 39.5%. At 18 months, the OS rate was 26.2% for the NALIRIFOX group, and 19.3% for the nab-paclitaxel and gemcitabine group.¹
• Trial met its secondary endpoint showing patients treated with NALIRIFOX had a statistically significant improvement in median PFS of 7.4 months versus 5.6 months for nab-paclitaxel and gemcitabine (HR 0.69 [95% CI 0.58–0.83]; p=0.0001).Error! Bookmark not defined.
• The objective response rate was 41.8% (36.8%-46.9%; 95% CI) for patients treated with the NALIRIFOX regimen versus 36.2% (31.4%-41.2%; 95% CI) for patients treated with nab-paclitaxel and gemcitabine.Error! Bookmark not defined.
• The safety profile of NALIRIFOX was consistent with the profiles of the treatment components. The most common Grade 3/4 treatment-emergent adverse events with more than 10% frequency in patients receiving NALIRIFOX versus nab-paclitaxel and gemcitabine included diarrhea (20.3% vs 4.5%), nausea (11.9% vs 2.6%), hypokalemia (15.1% vs 4.0%), anemia (10.5% vs 17.4%) and neutropenia (14.1% vs 24.5%).Error! Bookmark not defined.

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