US FDA grants rare paediatric disease designation to Race Oncology's RC220 bisantrene

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Race Oncology (ASX:RAC) has announced that the US FDA has extended Rare Paediatric Disease Designation (RPDD) to RC220 bisantrene for the treatment of paediatric subtypes of acute myeloid leukemia (AML).

The FDA previously granted the designation to RC110 bisantrene in 2018. The designation qualifies a sponsor to receive a Priority Review Voucher (PRV) from the US FDA at the time of marketing approval or authorisation for the drug in the paediatric rare disease area. 

The designation for paediatric AML may enable Race to be eligible to receive a PRV that can be redeemed for an accelerated six-month review of RC220 bisantrene or any other new drug application submitted to the US FDA. Granted PRVs may also transferred or sold to other companies for use in the same manner on the secondary market.

The reported purchase prices of PRVs to third parties on the open market have averaged more than US$100 million. Two PRVs were recently sold for US$110 million.

The company said it has been in discussion with Associate Professor Dr Himalee Sabnis at Emory University School of Medicine and a large dedicated international paediatric oncology cooperative group to explore undertaking a sponsored or investigator-initiated trial of RC220 bisantrene as a salvage treatment for paediatric AML patients.

Dr Sabnis is a key opinion leader in treating AML in paediatric patients.

Race chief medical officer Dr Michelle Rashford said, “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.”

CEO Dr Daniel Tillett added, “US FDA RPDD is incredibly valuable as not only does it offer eligibility for the award of a PRV, but the ability to work with passionate clinicians and regulators to bring help to children and adolescents facing an enormously challenging disease with few effective treatment options.”