The US Food and Drug Authority (FDA) has extended Rare...

The US Food and Drug Authority (FDA) has extended 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 pediatric 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 about 70 per cent of rare diseases are exclusively pediatric in onset, with 95 per cent 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.

RPDDs 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 pediatric rare disease area.

RAC says the RPDD for pediatric 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 $US100 million (range $US67.5 million to $350 million).

Two PRVs have been sold in recent times for $US110 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 pediatric AML study

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

Salvage treatment is given after a disease, such as cancer, fails to respond to other standard therapy treatments.

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

The company says several proposals from the international pediatric 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 pediatric 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 pediatric 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 pediatric 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.

New childhood cancer medicines needed

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 pediatric cancers,” she says.

“To be able to contribute to better treating childhood cancers like pediatric AML by collaboratively working with a dedicated international pediatric cooperative group would be very rewarding.”

RAC CEO Dr Daniel Tillett says the RPDD for its new RC220 bisantrene formulation for the treatment of pediatric 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,” he said.

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