A new experimental treatment for pancreatic cancer, ARB1002, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) and is expected to enter early clinical testing later this year.
The FDA reserves this status for treatments targeting rare diseases, or those affecting fewer than 200,000 people in the U.S. For the developer, Arbele, the designation provides financial and regulatory incentives, including a guaranteed seven years of market exclusivity if the therapy receives final approval.
“Orphan Drug Designation for ARB1002 marks a significant regulatory milestone for Arbele and reflects the critical need for new treatment options for patients with pancreatic cancer,” Linda Wu, PhD, chief development officer at Arbele, said in a company press release. “This designation reinforces our confidence in the therapeutic potential of ARB1002 and supports its continued advancement through clinical development.”
A targeted approach to killing cancer cells
ARB1002 belongs to a class of medicines known as antibody-drug conjugates (ADCs). A given antibody is able to stick to a specific molecular target with extremely high specificity. Antibodies produced by the immune system primarily target pieces of infectious bacteria and viruses; however, researchers can also engineer antibodies that target proteins on the surface of cancer cells. The goal of ADCs is to use antibodies to deliver a drug to cancer cells.
While natural antibodies fight off viruses and bacteria, the engineered antibodies in ARB1002 are specifically designed to target CDH17, a protein that is present in high concentrations on the surface of pancreatic cancer cells. By binding to CDH17, the antibody delivers a cytotoxic (cell-killing) drug specifically to the tumor, ultimately killing the cancerous cells.
Arbele is planning to launch a Phase 1 clinical trial testing ARB1002 in people with pancreatic cancer later this year.
Tony Wong, PhD, the company’s chief technology officer, noted that the designation “highlights CDH17 as a promising target in pancreatic cancer and validates Arbele’s ADC development strategy.” As the trial date approaches, the company plans to maintain a close dialogue with federal regulators to ensure the treatment moves through the pipeline as efficiently as possible.
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