https://www.theaustralian.com.au/business/*/us-fda-grants-argenica-orphan-drug-designation-to-treat-devastating-condition-in-newborns/news-story/88094bee862c26471ded87f332cdde64

US FDA grants Argenica orphan drug designation to treat ‘devastating condition’ in newborns

AGN has been granted US FDA Orphan Drug designation for its HIE treatment. Picture: Getty Images

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• 10:41AM NOVEMBER 16, 2023

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Argenica Therapeutics has achieved a key milestone, with the US Food and Drug Administration (FDA) granting orphan drug designation (ODD) for ARG-007 for the treatment of hypoxic ischaemic encephalopathy (HIE).

Perth-based Argenica Therapeutics (ASX: AGN) has announced its lead neuroprotective peptide candidate ARG-007 has been granted ODD status for the treatment of HIE, which is a type of newborn brain damage caused by oxygen deprivation and limited blood flow.

HIE may occur at any time before labour, during labour and delivery, or immediately following delivery.

AGN says the initial injury that is caused by a loss or reduction of oxygen supply is followed by progressive brain cell death due to excitotoxicity, oxidative stress, and inflammation.

The physiological effects resulting from the interruption to blood flow and/or oxygen in the brain can vary greatly depending on the length of time the disruption occurs, as well as its location.

While some children may only display mild effects, others will have severe permanent disability including cerebral palsy, cognitive impairment, or developmental delay.

Great need for HIE treatment

There is currently no therapeutic drug on the market to protect brain cells following cases of HIE.

Instead, therapeutic hypothermia (cooling the body to maintain a targeted temperature) is used.

This can prevent or minimise permanent brain damage in some babies, however, it can result in cardiovascular and respiratory complications.

AGN recently announced encouraging preclinical data on the potential of ARG-007 in reducing brain injury following HIE.

The data showed ARG-007 significantly reduces brain cell death in animal models of HIE.

The company is continuing to complete all required studies to progress into clinical trials in HIE.

FDA prioritises rare disease treatment

The FDA, which is the regulatory body in the world’s largest healthcare market, has been working to support development and evaluation of new treatments for rare diseases a key priority.

The FDA has authority to grant ODD to a drug or biological product to prevent, diagnose or treat a rare disease or condition.

ODD qualifies companies for incentives including:

• Tax credits for qualified clinical trials
• Exemption from user fees
• Potential seven years of market exclusivity after approval

AGN is developing novel therapeutics to reduce brain tissue death after stroke, other types of brain injury, and neurodegenerative diseases.

Argenica managing director Dr Liz Dallimore says the granting of ARG-007 in HIE forms a key pillar of its commercialisation strategy.

“The potential for extensive market exclusivity following approval of ARG-007 is an extremely compelling commercial driver for the company,” she says.

“We look forward to continuing to progress the development of ARG-007 as a treatment for HIE in newborn infants, in which there are currently no therapeutic drugs available to treat this devastating condition.”

This article was developed in collaboration with Argenica Therapeutics, a * advertiser at the time of publishing. This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.