FDA grants rare pediatric disease designation for QRX003 in peeling skin syndrome

Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) said in a press release dated September 8, 2026 that the U.S. Food and Drug Administration had granted Rare Pediatric Disease Designation to its lead asset, QRX003, for the treatment of Peeling Skin Syndrome. The company's Form 8-K covering the release was filed on September 10, 2026.

The designation is the second Rare Pediatric Disease Designation for QRX003. The FDA previously granted one for Netherton Syndrome. The release explains that the program is intended to encourage development of new therapies for serious and life-threatening diseases that primarily affect individuals under 18 years of age.

If a New Drug Application for QRX003 is approved, Quoin may qualify to receive a Priority Review Voucher. The release says the voucher can be redeemed to receive priority review for another marketing application, or it may be sold or transferred.

The development plan

Peeling Skin Syndrome is a rare genetic skin disease caused by loss-of-function variants of the corneodesmosin gene, CDSN. The release says it results in excessive shedding of the superficial layers of the epidermis. Patients generally suffer from severe pain and chronic pruritus, and there is currently no approved treatment.

Chief Executive Dr. Michael Myers said: "This will be the first formal study ever conducted in the U.S. for this disease under an open IND." The company says the IND is cleared and that it is preparing to start a Phase 2 study before the end of 2026. The study plans to enroll up to 12 pediatric and adult patients with Peeling Skin Syndrome in the United States and Europe.

Company background

Quoin's annual report for 2025, filed March 26, 2026, describes the company as a late-stage clinical specialty pharmaceutical company focused on rare and orphan diseases. The report says QRX003 is a topical lotion and that its active ingredient is a broad-spectrum serine protease inhibitor. It also says QRX003 is being developed as a potential treatment for Peeling Skin Syndrome, with the first subject treated in New Zealand. The company is expanding that study to include up to an additional five pediatric subjects.

The release describes QRX003 as the company's lead asset and says the designation reinforces its potential as a therapeutic candidate for a profoundly underserved pediatric population. Quoin's pipeline is focused on two platform products, QRX003 and QRX009.

The annual report also says QRX003 is currently being tested in seven pediatric Netherton Syndrome patients in investigator-initiated studies in Ireland, Austria, the Netherlands and New Zealand.

Financial position

The annual report shows research and development expense of $9.8 million for 2025, up from $3.6 million for 2024. It reports a net loss of $15.8 million for 2025 and $9.0 million for 2024. Cash from operations was negative $13.5 million in 2025 and negative $7.9 million in 2024.

What the designation does not mean

A rare pediatric disease designation is not a finding of benefit. The release reports no clinical data for QRX003 in Peeling Skin Syndrome, and the annual report describes a first subject treated in New Zealand, with the study being expanded by up to five pediatric subjects. The planned Phase 2 study has not started; the company says only that it expects to begin before the end of 2026. The priority review voucher would be available only if a New Drug Application is approved, and the release gives no date for submitting one.

The annual report says no products have been commercialized and no revenue has been generated. Cash used in operations was $13.5 million in 2025, and the quarterly report shows cash and cash equivalents of $3.8 million and short-term investments of $7.0 million at June 30, 2026.

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