Clinical stage biopharmaceutical company Race Oncology (ASX: RAC) has confirmed that the US Food and Drug Administration (FDA) has extended Orphan Drug Designation (ODD) to the company’s proprietary formulation of bisantrene, RC220.
US FDA ODD was first granted to Update Pharma Inc. in 2014, covering the RC110 formulation of bisantrene for the treatment of acute myeloid leukemia (AML). The ODD was transferred to Race in 2017 and this designation has been maintained by annual reporting of clinical and non-clinical activities involving bisantrene to the FDA.
The ODD programme was established by the US FDA under The Orphan Drug Act of 1983 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 USA. The Orphan Drug Act has led to the development and approval of 1240 new treatments and drugs.
ODD provides a wide range of benefits to sponsors of new treatments for orphan diseases 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$4.0m 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.
“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 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.”
Race Chief Medical Officer, Dr Michelle Rashford, said 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 programmes as we progress towards opening an FDA IND in 2025.
