Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Peeling Skin Syndrome

FDA’s second Rare Pediatric Disease designation for QRX003 adds potential PRV value and supports Quoin’s planned Phase 2 PSS trial.

Rhea-AI Summary

Quoin Pharmaceuticals (QNRX) received U.S. FDA Rare Pediatric Disease (RPD) Designation for its lead asset QRX003 in Peeling Skin Syndrome (PSS). This is the second RPD designation for QRX003, following an earlier designation for Netherton Syndrome, underscoring its potential for underserved pediatric patients. Under the RPD program, if an NDA for QRX003 is approved, Quoin may qualify for a Priority Review Voucher (PRV), which can be used for another application or sold or transferred. The company’s CEO stated that, if both indications ultimately yield PRVs, their aggregate non‑dilutive cash value could exceed $300 million at current trading values. With an IND cleared, Quoin expects to initiate a Phase 2 study in the second half of 2026, planning to enroll up to 12 pediatric and adult PSS patients in the U.S. and Europe, in what is described as the first formal PSS study in the U.S. under an open IND.

Positive

• Second RPD designation for QRX003, now covering both Peeling Skin Syndrome and Netherton Syndrome
• If approved in both indications, potential for two PRVs that management estimates could exceed $300 million in aggregate non‑dilutive value at current trading levels
• IND cleared and Phase 2 PSS study expected to start in 2H 2026 with up to 12 patients in the U.S. and Europe

Negative

• None.

Key Figures

RPD designations: 2 Potential PRVs: 2 Potential PRV value: In excess of $300 million +2 more

RPD designations

2

QRX003, including the new Peeling Skin Syndrome designation

Potential PRVs

2

Conditional on NDA approval for QRX003 indications

Potential PRV value

In excess of $300 million

Aggregate non-dilutive cash value cited by management

Phase 2 timing

2H 2026

Planned Peeling Skin Syndrome study initiation

Planned enrollment

Up to 12 patients

Pediatric and adult Peeling Skin Syndrome patients in the U.S. and Europe

Previous Fda approval Reports

1 past event · Latest: Jun 24

Same Type 1 event

1. Jun 24

   FDA designation

   24h Move

   +4.7%

   FDA granted QRX003 Rare Pediatric Disease Designation for Netherton Syndrome, creating PRV eligibility.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

rare pediatric disease designation, new drug application, priority review voucher

3 terms

rare pediatric disease designation regulatory

"FDA has granted Rare Pediatric Disease (RPD) Designation"

A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.

new drug application regulatory

"If a New Drug Application (NDA) for QRX003 is approved"

A new drug application is a formal request submitted to government regulators seeking approval to market a new medicine. It is like a detailed proposal that shows the drug has been tested for safety and effectiveness. For investors, receiving approval signals that the drug may soon become available for sale, potentially leading to revenue growth and impacting the company's value.

priority review voucher regulatory

"Quoin may qualify to receive a Priority Review Voucher (PRV)"

A priority review voucher is a transferable regulatory incentive that lets a company move a future drug or device application to the front of the review line, shortening the review period by several months. For investors it matters because the voucher can speed up market access for a high-value product or be sold to other companies for significant cash, acting like a tradable fast-pass that can accelerate revenue or create immediate financial upside.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• Second Rare Pediatric Disease (RPD) Designation for QRX003
• FDA Previously Granted RPD Designation for QRX003 in Netherton Syndrome
• If a New Drug Application (NDA) for QRX003 Is Approved for Peeling Skin Syndrome, Quoin May Receive a Freely Tradable Priority Review Voucher (PRV)
• Quoin Expects to Initiate Phase 2 Study in 2H 2026; Study Plans to Enroll up to 12 Pediatric and Adult Peeling Skin Patients in the U.S. and Europe
• There are Currently No Approved Treatments for Peeling Skin Syndrome

ASHBURN, Va., Sept. 08, 2026 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (“Quoin” or the “Company”), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the Company’s lead asset, QRX003, for the treatment of Peeling Skin Syndrome (PSS).

The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. This is the second RPD designation granted for QRX003, following the previously granted RPD designation for Netherton Syndrome.

The FDA’s Rare Pediatric Disease Designation program is intended to encourage the development of new therapies for serious and life-threatening diseases that primarily affect individuals under 18 years of age. If a New Drug Application (NDA) for QRX003 is approved, Quoin may qualify to receive a Priority Review Voucher (PRV), which can be redeemed to receive priority review for another marketing application or may be sold or transferred.

“We are very pleased to announce the receipt of Rare Pediatric Disease Designation for QRX003 for Peeling Skin Syndrome. This designation means that Quoin could potentially receive two PRVs, which, given the current trading value of PRVs, could have an aggregate non-dilutive cash value in excess of $300 million,” said Dr. Michael Myers, Chief Executive Officer of Quoin Pharmaceuticals. “With the IND cleared by FDA, the Quoin team is preparing to initiate the Phase 2 clinical study before the end of this year with plans to enroll up to 12 pediatric and adult Peeling Skin patients in the U.S. and Europe. This will be the first formal study ever conducted in the U.S. for this disease under an open IND.”

About Peeling Skin Syndrome (PSS)
Generalized inflammatory peeling skin syndrome (PSS) is a rare autosomal recessive genodermatosis caused by loss-of-function disease-causing variants of the corneodesmosin gene (CDSN), resulting in excessive shedding of the superficial layers of the epidermis. Patients generally suffer from a variety of conditions including severe pain and chronic pruritus (itch). There is currently no approved treatment for PSS.

About Quoin Pharmaceuticals Ltd.
Quoin Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities, and care teams. Quoin's innovative pipeline is focused on two key platform products, QRX003 and QRX009, that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Pachyonychia Congenita, Gorlin Syndrome and Tuberous Sclerosis Complex, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibromas and others. For more information, visit: www.quoinpharma.com or LinkedIn for updates.

Cautionary Note Regarding Forward Looking Statements
The Company cautions that statements in this press release that are not a description of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” “look forward to,” and “will,” among others. All statements that reflect the Company’s expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: the potential of QRX003 as a therapeutic candidate for Peeling Skin Syndrome and a profoundly underserved pediatric population, Quoin’s eligibility to receive Priority Review Vouchers upon approval of a New Drug Application for QRX003, including the potential to receive two PRVs with an aggregate non-dilutive cash value in excess of $300 million; the initiation of a Phase 2 clinical study for Peeling Skin Syndrome before the end of 2026 with plans to enroll up to 12 pediatric and adult patients in the U.S. and Europe; and Quoin’s belief that its products in development collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Pachyonychia Congenita, Gorlin Syndrome and Tuberous Sclerosis Complex, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibromas and others.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company’s ability to pursue its regulatory strategy; the Company’s ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company’s ability to complete clinical trials on time and achieve desired results and benefits as expected; and other factors discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law.

For further information, contact:

Quoin Pharmaceuticals Ltd.
Michael Myers, Ph.D., CEO
mmyers@quoinpharma.com

Investor Relations
PCG Advisory
Jeff Ramson
jramson@pcgadvisory.com
(646) 863-6341

FAQ

What does Rare Pediatric Disease (RPD) Designation provide for Quoin and QRX003?

The RPD program is intended to encourage development of therapies for serious or life‑threatening diseases that primarily affect individuals under 18. If an NDA for QRX003 is approved in an RPD indication, Quoin may qualify to receive a Priority Review Voucher, which can be redeemed for priority review of another marketing application or sold or transferred.

How many Priority Review Vouchers could Quoin potentially receive from QRX003?

QRX003 now has RPD designation for both Peeling Skin Syndrome and Netherton Syndrome. The CEO stated that this means Quoin could potentially receive two PRVs if NDAs for QRX003 in both indications are ultimately approved.

What are the key design elements of the planned Phase 2 PSS study?

Quoin expects to initiate a Phase 2 clinical study of QRX003 in Peeling Skin Syndrome before the end of 2026. The trial plans to enroll up to 12 pediatric and adult PSS patients in the U.S. and Europe and is described as the first formal study in the U.S. for this disease under an open IND.

What is Peeling Skin Syndrome (PSS)?

Generalized inflammatory Peeling Skin Syndrome is a rare autosomal recessive skin disorder caused by loss‑of‑function variants in the corneodesmosin (CDSN) gene, leading to excessive shedding of the superficial epidermal layers. Patients often experience severe pain and chronic pruritus, and there is currently no approved treatment for PSS.

What other indications is Quoin targeting with its pipeline?

Quoin is developing QRX003 and QRX009 for a range of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Pachyonychia Congenita, Gorlin Syndrome, Tuberous Sclerosis Complex, Microcystic Lymphatic Malformations, Venous Malformations and Angiofibromas, among others.