FDA Orphan Drug Status Granted to Anti-CDH17 ADC in Pancreatic Cancer

The FDA has granted orphan drug designation (ODD) to ARB1002, an investigational anti-CDH17 antibody-drug conjugate, for the treatment of pancreatic cancer.¹

With pancreatic cancer’s aggressive nature and poor prognosis, patients with this disease continue to grapple with limited therapeutic options and low long-term survival rates, underscoring the need for novel and more effective treatments.² While the landscape of targeted therapies for pancreatic cancer has expanded in recent years, the field has encountered continued difficulties targeting various mutated genes, and few agents have demonstrated durable benefit, especially in the first line.

The ODD, awarded to agents that promise to treat rare diseases or conditions, will provide sponsor Arbele with incentives including tax credits for clinical trials, user fee exemptions, and a potential 7 years of market exclusivity following approval, all of which are intended to support the development pathway to ultimately bring ARB1002 to patients.³

“[ODD] for ARB1002 marks a significant regulatory milestone… and reflects the critical need for new treatment options for patients with pancreatic cancer,” said Linda Wu, chief development officer of Arbele, in a news release.¹ “This designation reinforces our confidence in the therapeutic potential of ARB1002 and supports its continued advancement through clinical development.”

About ARB1002

CDH17 is a cell-surface adhesion protein that is highly expressed in intestinal epithelial cells and has therefore generated interest as a therapeutic target for gastrointestinal cancers.⁴ ARB1002 consists of an anti-CDH17 monoclonal antibody conjugated to a potent cytotoxic agent. Its specificity to CDH17 allows for targeted delivery of the cytotoxic payload to tumor cells, thereby limiting off-target effects.

Stage of Development

According to the sponsor, investigational new drug-enabling studies are underway in preparation to advance ARB1002 into a phase 1 clinical trial, to be launched later in 2026. An abstract detailing results from one of these studies will be presented imminently during the 2026 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium from January 8 to 10.

“We look forward to continued engagement with the FDA as we progress ARB1002,” added Tony Wong, chief technology officer of Arbele, in the release.¹

REFERENCES

1. Arbele receives FDA orphan drug designation for ARB1002, an anti-CDH17 antibody–drug conjugate for the treatment of pancreatic cancer. News release. Arbele. January 7, 2026. Accessed January 8, 2026. https://tinyurl.com/thrmdc2r

2. Hayat U, Croce PS, Saadeh A, et al. Current and emerging treatment options for pancreatic cancer: a comprehensive review. J Clin Med. 2025;14(4):1129. Published 2025 Feb 10. doi:10.3390/jcm14041129

3. Designating an orphan product: drugs and biological products. US Food & Drug Administration. Updated August 12, 2024. Accessed January 8, 2026. https://tinyurl.com/5n77pu2w

4. Jacobsen F, Pushpadevan R, Viehweger F, et al. Cadherin-17 (CDH17) expression in human cancer: A tissue microarray study on 18,131 tumors. Pathol Res Pract. 2024;256:155175. doi:10.1016/j.prp.2024.155175