Race Oncology secures US FDA rare paediatric disease designation for RC220 bisantrene

Race Oncology Limited (ASX: $RAC) has announced that the United States Food and Drug Administration (US FDA) has extended Rare Paediatric Disease Designation (RPDD) to RC220 bisantrene for the treatment of childhood subtypes of AML. This designation provides eligibility for Race to receive a Priority Review Voucher (PRV) upon marketing approval, potentially offering significant financial opportunities.

Executive commentary on US FDA rare paediatric disease designation

Race Chief Medical Officer, Dr Michelle Rashford, expressed, 'There is a need for new medicines designed to treat these rare childhood cancers which can be devastating for families. The US government has created incentives like the Priority Review Voucher scheme to encourage companies to invest in research and clinical studies in paediatric cancers. To be able to contribute to better treating childhood cancers like paediatric AML by collaboratively working with a dedicated international paediatric cooperative group would be very rewarding.'

Summary of US FDA rare paediatric disease designation for RC220 bisantrene

The US FDA has extended Rare Paediatric Disease Designation to Race Oncology's RC220 bisantrene, potentially allowing the company to receive a Priority Review Voucher (PRV) upon marketing approval. This designation signifies a significant opportunity for Race to contribute to the treatment of rare childhood cancers, particularly paediatric AML. The company is in discussions with a renowned paediatric oncology cooperative group to explore undertaking a sponsored or investigator-initiated trial of RC220 bisantrene as a salvage treatment for paediatric AML patients. Race plans to submit a US FDA Investigational New Drug application for RC220 bisantrene in 2025, indicating a clear path forward for the potential development of this treatment. The extension of the RPDD program is anticipated, providing a positive outlook for Race's efforts in addressing the unmet medical needs of paediatric AML patients.