What is Netherton Syndrome?

Netherton Syndrome is a rare genetic disorder that primarily affects newborns, impacting approximately 1 in 200,000 births globally. It leads to severe skin inflammation and dehydration,often requiring extended hospital stays.

What is the Rare Pediatric Disease Designation?

The FDA's Rare Pediatric Disease Designation aims to incentivize the development of new treatments for serious and life-threatening diseases that predominantly affect individuals under the age of 18.

QRX003 is quoin Pharmaceuticals' lead asset,currently in late-stage clinical development,designed as a potential therapeutic option for Netherton Syndrome.

What are the potential benefits of receiving a Rare Pediatric Disease Designation?

If QRX003 receives FDA approval, Quoin Pharmaceuticals may be eligible for a Priority Review Voucher (PRV). This voucher can expedite the review process for another marketing application or be sold to another company.

What other designations has QRX003 received?

In May 2025, the European Medicines Agency (EMA) granted Orphan Drug Designation to QRX003, further recognizing its potential in treating Netherton Syndrome.

What is Quoin Pharmaceuticals focused on?

Quoin Pharmaceuticals is dedicated to creating and commercializing treatments for rare and orphan diseases, addressing unmet medical needs for patients and their families.

Health

Quoin Pharma’s QRX003 Receives FDA Rare Pediatric Disease Designation for Netherton Syndrome

by DrMichaelLee June 24, 2025

written by DrMichaelLee June 24, 2025

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Quoin Pharma’s QRX003 Gains FDA Rare Pediatric Disease Designation for Netherton Syndrome

ASHBURN, Va. – Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) announced today that the U.S.Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to QRX003, its primary drug candidate, for the treatment of Netherton Syndrome. This designation underscores the potential of QRX003 to address a critical unmet need within the pediatric population suffering from this severe genetic condition.

FDA Recognition for Netherton Syndrome Treatment

The FDA

DrMichaelLee

Dr. Michael Lee – Health Editor Dr. Michael Lee is a physician, medical writer, and Health Editor for World Today News. He translates complex health topics into clear, practical advice and leads our coverage of medical breakthroughs, public health, and wellness.

Quoin Pharma’s QRX003 Receives FDA Rare Pediatric Disease Designation for Netherton Syndrome

Quoin Pharma’s QRX003 Gains FDA Rare Pediatric Disease Designation for Netherton Syndrome

FDA Recognition for Netherton Syndrome Treatment

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