BRIEF—Rare pediatric disease and orphan drug designations for retinoblastoma candidate

Privately-held UK-based company Oxular has announced that it has received both Rare Pediatric Disease and Orphan Drug designations from the US Food and Drug Administration (FDA) for OXU-003, the firm’s proprietary drug in development for the treatment of retinoblastoma.

This rare form of eye cancer usually develops in early childhood, typically before the age of five. It is often curable when diagnosed early. However, if it is not treated promptly, this cancer can spread beyond the eye to other parts of the body. This advanced form of retinoblastoma can be life-threatening.

OXU-003 has been shown to be effective as a stand-alone therapy in pre-clinical models and is complementary with other agents currently used to treat retinoblastoma. The company expects the drug to enter human clinical trials and generate data within the next two years.