Revive Therapeutics Ltd (CSE:RVV, OTCQB:RVVTF) has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its Bucillamine treatment for the prevention of ischemia-reperfusion injury (IRI) during liver transplantation.
The FDA grants ODD status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. It defines rare diseases as those affecting fewer than 200,000 people in the US at any given time.
"The ODD validates our strategy in pursuing novel uses of Bucillamine for rare disorders and life-threatening conditions, including infectious diseases and preventing IRI during other organ transplantations,” Revive CEO Michael Frank said in a statement.
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Revive noted that currently there are no approved treatments available for IRI. Liver ischemia-reperfusion injury is a major complication of liver transplantation and is one of the leading causes for post-surgery hepatic dysfunction leading to an increased risk of post-operative morbidity and mortality.
“According to the United Network for Organ Sharing there were 8,906 liver transplants in 2020 and at the time of the ODD submission there were 11,664 on the waiting list for a liver transplant,” Revive said.
“Although many therapeutic strategies have been shown to be effective in controlled experimental models, most have yielded equivocal results in clinical practice or have yet to reach human clinical trials,” the company added.
Revive said it believes the use of Bucillamine during liver transplantation has the potential to be a safe and effective approach to address the unmet medical need for a novel strategy to limit or prevent IRI.
Bucillamine, a cysteine derivative that contains two donatable thiol groups, in the context of IRI is capable of replenishing the thiol group in glutathione, thereby reactivating this endogenous defense against oxidant injury, Revive said.
In addition, it noted that Bucillamine appears to have anti-inflammatory effects unrelated to its antioxidant effect. Bucillamine has the potential to address the shortage of quality organs by reducing the susceptibility to IRI of steatotic livers thereby making these livers available for transplants. The drug also has the potential to improve graft function and patient outcome by preventing or lessening IRI, it added.
The company said ODD would qualify Bucillamine for certain benefits and incentives, including seven years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain clinical drug testing costs, activities, eligibility for orphan drug grants, and the waiver of the FDA New Drug Application filing fee of approximately US$2,400,000.
Revive Therapeutics is focused on the research and development of therapeutics for infectious diseases and rare disorders and aims to take advantage of several regulatory incentives awarded by the FDA, such as orphan drug, fast track, breakthrough therapy and rare pediatric disease designations.
Contact the author at stephen.gunnion@proactiveinvestors.com