• US FDA extends Rare Paediatric Disease Designation (RPDD) to Race Oncology’s novel RC220 bisantrene drug
  • RPDD gives RC220 eligibility to receive a Priority Review Voucher (PRV) upon marketing approval
  • PRV’s can be transferred or sold to other parties with sales averaging more than US$100 million


Special Report: The US FDA extends Rare Paediatric Disease Designation (RPDD) to Race’s oncology drug.



Race Oncology (ASX:RAC) says the RPDD extension for its new RC220 bisantrene formulation to treat AML in children comes as interest grows in the drug from imminent paediatric oncologists globally.

RPDD is granted for new treatments of serious or life-threatening diseases which affect fewer than 200,000 people in the US and which primarily affect individuals less than 18 years of age.

RAC says ~70% of rare diseases are exclusively paediatric in onset, with 95% of rare diseases having no approved treatments.  The company was previously granted RPDD by the US FDA for the RC110 formulation of bisantrene in 2018.


RPDD’s sought after by biotechs

RPDD qualifies a sponsor eligible to receive a Priority Review Voucher (PRV) from the US FDA at the time of marketing approval or authorisation for a drug in the paediatric rare disease area.

RAC says the RPDD for paediatric AML may enable the biotech 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 be 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 (range US$67.5 million to $350 million). Two PRVs have been sold in recent times for US$110 million.

June has been a good month for RAC, which earlier this month announced The FDA had extended orphan drug designation (ODD) to the company for bisantrene RC220 for AML treatment.


Plans for investor-led paediatric AML study

RAC has been in talks with Associate Professor Dr Himalee Sabnis from the department of paediatrics at Emory University School of Medicine in Atlanta and a large international paediatric oncology cooperative group to undertake a sponsored or investigator-initiated trial of RC220 bisantrene as a salvage treatment for paediatric AML patients.

Salvage treatment is given after a disease, such as cancer, does not respond to other standard therapy treatments.

RAC says Dr Sabnis is a world-leading, key opinion leader in the treatment of AML in paediatric patients.

The company says several proposals from the international paediatric cooperative oncology group and Dr Sabnis have been received and possible options for conducting the trial have been discussed.

Options include a fully RAC sponsored trial, a hybrid approach where costs and data would be shared, or a fully investigator sponsored trial under the management of the paediatric cooperative oncology group where the data could later be made available to Race.

No final agreement on the best approach has been reached, but RAC says it is highly encouraged by the continued and ongoing interest from both the paediatric cooperative oncology group and Dr Sabnis.

RAC says before starting any trial in the US and other countries, a FDA Investigational New Drug (IND) for RC220 bisantrene must be in place.

An agreement must also be reached with the FDA and the international paediatric collaborative oncology group on the trial design and funding model must be established.

RAC plans to submit a US FDA IND application for RC220 bisantrene in 2025.


Need for new medicines to treat childhood cancer

RAC chief medical officer Dr Michelle Rashford says 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,” she says.

“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.”

RAC CEO Dr Daniel Tillett says the RPDD for its new RC220 bisantrene formulation for the treatment of paediatric subtypes of AML will help open doors to advance the drug.

“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.”


This article was developed in collaboration with Race Oncology, a Stockhead advertiser at the time of publishing.  

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.