FDA and EMA Grant Orphan Drug Status to Novel Imaging Drug for Pancreatic Cancer

The FDA and European Medicines Agency (EMA) have awarded orphan drug designation to CPI-008 (cRGD-ZW800-1), a novel integrin-targeted, zwitterionic imaging agent, for margin detection of pancreatic cancer during surgery.¹

The FDA’s orphan drug status is given to agents that seek to address rare diseases and conditions that affect fewer than 200,000 patients in the US.² The designation, which will support the continued development of CPI-008 in pancreatic cancer, paves the way for more precise and effective surgical treatments in this rare, challenging disease.

“Receiving orphan [drug] designation from both the FDA and the EMA is a profoundly important milestone for Curadel, granting us valuable incentives to fuel our development efforts,” said John V. Frangioni, MD, PhD, CEO of Curadel Pharma, in a news release.¹ “As a pioneering company working to introduce significant advances in surgical imaging, the efficiencies of fee exemptions, credits, along with the potential for market exclusivity are vital tools to help us smartly deploy our resources and focus on delivering value to the surgical community.”

CPI-008 and the Imaging Dilemma in Pancreatic Cancer

A major obstacle in pancreatic cancer lies in the deep anatomical position of the pancreas, which poses challenges not only in initial diagnosis, but also during surgical removal. Current limitations of imaging tools may leave some cancer cells undetected during pancreatectomies, hindering complete resection of the tumor.

Near-infrared (NIR) fluorescence imaging is a surgical technique that can offer real-time visual guidance to surgeons by helping illuminate structures during surgery, allowing for improved distinction between malignant and benign tissue. CPI-008 is a fluorescent, zwitterionic agent that can be injected intravenously to enhance tumor cell visualization and detection in the pancreas by binding to integrins overexpressed on tumor cells and tumor-associated vascular endothelium.³ Its development, which is ongoing in multiple cancers including pancreatic cancer, head and neck cancer, and colorectal cancer, is a promising approach that shows potential to optimize surgical precision and overall outcomes.

About the FLUOPANC Trial

The designation is supported by positive findings from the single-center phase 2 FLUOPANC study (NCT05518071), completed in May 2024. This study demonstrated CPI-008’s imaging capabilities in 20 patients with pancreatic ductal adenocarcinoma or extrahepatic cholangiocarcinomas with or without neoadjuvant treatment.^3,4

This dose-ranging and dose-selection study was designed to evaluate the feasibility, safety, and tolerability of CPI-008 in 4 dose-finding cohorts and an expansion cohort. Specifically, the primary end point of the study was visualization of the primary tumor using CPI-008 and NIR-fluorescence imaging, which was measured using the tumor-to-background ratio both in vivo and ex vivo. Key secondary end points included number and grade of treatment-emergent adverse events; optimal dose and dose interval of CPI-008; and tumor-positive margins, lymph nodes, and metastases detected with NIR-fluorescence imaging.

In the study, patients received a single bolus injection of CPI-008 between 2 and 24 hours before surgery. Tumors and related structures were assessed in vivo during surgery; following surgery, assessments were performed ex vivo on resected tissue.

REFERENCES

1. FDA and EMA grant orphan drug designation for Curadel’s CPI-008, a targeted zwitterionic imaging drug for pancreatic cancer. News release. Curadel Pharma. January 7, 2026. Accessed January 7, 2026. https://tinyurl.com/bpa3f5pp

2. Designating an orphan product: drugs and biological products. US Food & Drug Administration. Updated August 12, 2024. Accessed January 7, 2026. https://tinyurl.com/5n77pu2w

3. FLUOPANC-trial - Fluorescence-guided surgery of pancreatic and bileduct tumors using cRGD-ZW800-1 (FLUOPANC). ClinicalTrials.gov. Updated June 24, 2024. Accessed January 7, 2026. https://clinicaltrials.gov/study/NCT05518071

4. Van Dam MA, van Ravens T, Burggraaf J, et al. Optical navigation using tumour-targeted near-infrared (NIR) fluorescence in pancreatic surgery: a phase II feasibility trial of a novel integrin-targeting fluorescent probe cRGD-ZW800-1. Eur J Surg Oncol. 2024;50:108816. doi:10.1016/j.ejso.2024.108816