Race Oncology’s bisantrene RC220 AML treatment earns coveted FDA orphan drug designation

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By Colin Hay - 
RAC FDA orphan drug designation Bisantrene

Race Oncology (ASX: RAC) has achieved a significant breakthrough with the US Food and Drug Administration (FDA) awarding orphan drug designation (ODD) to the company’s acute myeloid leukemia (AML) candidate bisantrene RC220.

The coveted designation qualifies Race for various benefits including tax credits, fee exemptions, market exclusivity and regulatory assistance from the FDA throughout the drug development process.

Rigorous evaluation

Bisantrene is a small molecule anthracene-based chemotherapeutic that has been evaluated in more than 46 clinical trials and 70 peer-reviewed publications, demonstrating efficacy and a well-characterised safety profile on approximately 1,800 patients.

RC220 is the company’s flagship bisantrene formulation for peripheral infusion.

Race revealed in May 2024 that trials had found that bisantrene and decitabine used together offered significantly improved cancer cell killing across a broad panel of 143 tumour cell lines compared to when either drug was used alone.

These results supported the use of decitabine in combination with bisantrene as a potential treatment for many cancers including solid tumour types such as lung, head and neck, prostate, pancreas and breast.

The bisantrene/decitabine combination will be further explored in a proposed phase 1/2 investigator-initiated AML clinical trial.

ODD benefits

The ODD program was established by the FDA to promote the development of products that aid in the diagnosis and/or treatment of rare diseases or conditions, defined as those affecting fewer than 200,000 people within the US and has so far led to the development and approval of 1,240 new treatments and drugs.

ODD confers a wide range of benefits to sponsors of new treatments for orphan diseases including 7-year US marketing exclusivity, 25% federal tax credit for expenses incurred from clinical research conducted within the US that may be applied towards up to 20 years of future taxes, the waiving of fees with a value of $6.2 million as of 2024, qualification to apply for research grants to support clinical studies of orphan drugs and eligibility to receive additional ongoing regulatory assistance and guidance from the FDA.

Novel treatment

“Race continues to advance bisantrene as a novel treatment for AML,” said chief executive officer Dr Daniel Tillett.

“However, being able to leverage the additional regulatory and guidance support from the FDA that ODD provides is very welcome.”

Chief medical officer Dr Michelle Rashford added, “ODD designation is a major asset beyond AML as it enables Race to work closely and constructively with the FDA on all of our RC220 bisantrene clinical programs as we progress towards opening an FDA investigational new drug [application] in 2025.”