The United States Food and Drug Administration (US FDA) has extended the rare paediatric disease designation (RPDD) of the novel Race Oncology Ltd (ASX:RAC, OTC:RAONF) drug RC220 bisantrene for the treatment of paediatric subtypes of Acute Myeloid Leukaemia (AML).
This designation was previously granted to RC110 bisantrene in 2018.
Rare disease
RPDD is awarded for new treatments of serious or life-threatening diseases affecting fewer than 200,000 people in the US, primarily those under 18 years of age.
Around 70% of rare diseases are exclusively paediatric in onset, with 95% having no approved treatments.
The designation makes RC220 eligible for a priority review voucher (PRV) upon marketing approval, which can be transferred or sold.
CEO Dr Daniel Tillett said: "US FDA RPDD is incredibly valuable as it offers eligibility for the award of a PRV, and 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."
Historically, PRVs have sold for an average of over US$100 million. Recent sales have ranged from US$67.5 million to US$350 million, with two recent transactions at US$110 million.
The company is exploring a sponsored or investigator-initiated trial of RC220 as a salvage treatment for paediatric AML patients.
Discussions are underway with Associate Professor Himalee Sabnis of the Department of Pediatrics at Emory University School of Medicine, and a large international paediatric oncology cooperative group. Dr Sabnis, a leading expert in paediatric AML, expressed keen interest in bisantrene.
Race chief medical officer Dr Michelle Rashford highlighted the importance of new treatments for rare childhood cancers: "There is a need for new medicines designed to treat these rare childhood cancers which can be devastating for families.
Incentive for R&D
“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.”
To be awarded a PRV by the US FDA, Race must demonstrate that RC220 bisantrene significantly improves specific paediatric AML clinical outcomes, either by increasing patient survival or reducing serious side effects such as heart damage.
Given the ultra-rare nature of paediatric AML (less than seven cases per million children under 15), any trial would likely be modest in size, involving fewer than 30 patients.
Although the RPD program is scheduled to end in 2026, it is widely expected to be extended by the US Congress, having consistently received bipartisan support.