A neuroprotective drug developed in Western Australia has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration for the treatment of hypoxic ischaemic encephalopathy (HIE) in newborn term infants.
HIE is a form of brain injury resulting from babies being deprived of oxygen before, during or immediately after birth. Some may only display mild effects while others will have severe permanent disability including cerebral palsy, cognitive impairment, or developmental delay.
HIE contributes to one of the leading causes of death and disability in children under two.
Achieving RPDD status from the FDA is a significant milestone for WA-based biotechnology company Argenica Therapeutics which is developing novel therapeutics to reduce brain tissue death after stroke and other types of brain injury and neurodegenerative diseases.
Argenica’s lead neuroprotective peptide candidate ARG-007 has been shown to improve outcomes in preclinical models of stroke, traumatic brain injury and HIE.
Now in a Phase 2 clinical trial for acute ischaemic stroke, this potentially revolutionary drug has been developed through research led by Professor Bruno Meloni and Clinical Professor Neville Knuckey (Perron Institute and The University of Western Australia).
Leading the HIE research at the Perron Institute is Dr Adam Edwards, a Postdoctoral researcher in the Institute’s Stroke Research group and Argenica’s Neonatal Scientific and Regulatory Advisor.
“Supporting the development and evaluation of new treatments for rare diseases in children is a key priority for the FDA,” said Dr Liz Dallimore, Argenica’s Managing Director.
“This further validation on the potential of ARG-007 as a prospective treatment for HIE through the granting of a Rare Pediatric Disease Designation is extremely encouraging.
“There are currently no therapeutic drugs available to treat this devastating condition.
“The RPDD will provide Argenica Therapeutics with potential significant upside at the end of a clinical program in HIE to receive a priority review to get the drug on the market quickly, or the option to on-sell to a third party.
“We look forward to continuing to progress the development of ARG-007 as a treatment for HIE in newborn infants.”