FDA Grants ODD for Esophageal, Gastroesophageal Junction, Pancreatic Cancer Treatment

Apexigen announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation status to APX005M for the treatment of esophageal and gastroesophageal junction cancer and for the treatment of pancreatic cancer.

"These orphan drug designations are important regulatory milestones for Apexigen in its mission to treat cancer patients and for APX005M, which has the potential to meaningfully impact the standard of care across multiple types of solid tumors. We are evaluating APX005M in a broad clinical program that includes more than 10 clinical trials in various indications and therapeutic combinations," said Xiaodong Yang, MD, PhD, Chief Executive Officer of Apexigen.

The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor for various development incentives, including tax credits for qualified clinical testing, up to seven years of marketing exclusivity for the orphan indication and waiver of certain FDA fees.

APX005M is a novel, humanized monoclonal antibody that stimulates the anti-tumor immune response. APX005M targets CD40, a co-stimulatory receptor that is essential for activating both innate and adaptive immune systems. Binding of APX005M to CD40 on antigen presenting cells (i.e., dendritic cells, monocytes and B-cells) initiates a multi-faceted immune response bringing multiple components of the immune system (e.g., T cells, macrophages) to work in concert against cancer. APX005M is currently in Phase 2 clinical development for the treatment of cancers such as pancreatic cancer, esophageal and gastroesophageal junction cancers, melanoma, non-small cell lung cancer, rectal cancer and sarcoma in various combinations with immunotherapy, chemotherapy, radiation therapy or a cancer vaccine.