Race Oncology Ltd (ASX:RAC, OTC:RAONF) has welcomed the extension of the company’s proprietary formulation of bisantrene, RC220, as an Orphan Drug Designation (ODD) by the US Federal Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML).
ODD was originally awarded to Update Pharma Inc. in 2014, covering the RC110 formulation of bisantrene, and was transferred to RAC and then maintained by annual reporting of clinical and non-clinical activities involving bisantrene to the FDA.
The ODD program was established to promote the development of drugs, biologics, devices or medical foods 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 – it has led to the development and approval of 1,240 new drugs and treatments.
Welcoming the extension, investors have sent Race Oncology shares 7.6% higher intra-day on the ASX to a new 12-month high of A$1.92.
Pathway to collaboration with FDA
“I wish to thank the clinical team at Race for their foresight in seeking to extend FDA Orphan Drug Designation to our new RC220 bisantrene formulation,” Race Oncology CEO Dr Daniel Tillett said.
“Race continues to advance bisantrene as a novel treatment for AML, however, being able to leverage the additional regulatory and guidance support from the FDA that ODD provides is very welcome.”
The ODD provides several benefits, including:
- 7-year US marketing exclusivity for approved orphan products.
- 25% federal tax credit for expenses incurred from clinical research conducted within the United States. The tax credits may be applied towards up to 20 years of future taxes.
- Waiver of fees under the Prescription Drug User Fee Act (PDUFA) with a value of US$4.0 million as of 2024.
- Qualification to apply for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies of orphan drugs.
- Eligibility to receive additional regulatory assistance and guidance from the FDA in the design of an overall drug development plan.
“Orphan Drug 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 IND in 2025,” Race Oncology chief medical officer Dr Michelle Rashford said.