Quoin Targets World’s First Approval for Netherton Syndrome Therapy in Saudi Arabia

Quoin Pharmaceuticals has announced the submission of an application for Breakthrough Medicine Designation to the Saudi Food and Drug Authority (SFDA) for QRX003, its lead late-stage topical therapy under development for Netherton Syndrome.¹ If granted, this designation could significantly accelerate regulatory review and enable QRX003 to become available for sale and reimbursement in Saudi Arabia as early as the second half of 2026. It would then become the first approved treatment worldwide for this rare genodermatosis.

Quoin has also established a regional distribution partnership with Genpharm covering Saudi Arabia and other Middle East and North Africa (MENA) countries. If Breakthrough Medicine Designation is granted by the SFDA, this partnership could facilitate rapid access for patients in Saudi Arabia, potentially ahead of approvals in the US or Europe.

“Filing for Breakthrough Medicine Designation with the SFDA marks a historic milestone for both Quoin and the Netherton Syndrome community,” said Michael Myers, PhD, Chief Executive Officer of Quoin. “If granted, it is possible that QRX003 could be available for sale and reimbursement in Saudi Arabia in the second half of this year. This would make QRX003 the first ever approved treatment anywhere in the world for this devastating disease. We look forward to working with our commercial partner in the region to make QRX003 available to Netherton patients in Saudi Arabia as expeditiously as possible, if the designation is granted.”¹

Netherton Syndrome is a severe, lifelong inherited disorder of skin barrier function characterized by generalized erythroderma, ichthyosis linearis circumflexa, hair shaft abnormalities, and profound atopic manifestations. Patients often experience recurrent infections, failure to thrive, and substantial impairment in quality of life beginning in infancy. Despite the seriousness of the disease, there are currently no approved therapies, and management relies largely on supportive care.

The SFDA program is designed to expedite the development, assessment, and potential approval of medicines that target serious or life-threatening conditions for which existing treatment options are inadequate or nonexistent. Eligibility criteria include the potential for substantial clinical benefit over current approaches, an acceptable benefit–risk profile, and the absence of prior regulatory approval at the time of application. Quoin has indicated that QRX003 meets all of these requirements, based on the severity of Netherton Syndrome, the lack of approved therapies, and encouraging clinical data generated to date.

QRX003 is a 4% topical lotion intended for whole-body application and is being evaluated as a disease-modifying therapy rather than purely symptomatic treatment. The product is currently in 2 late-stage, whole-body pivotal clinical trials enrolling patients with Netherton Syndrome. These studies are designed to assess both efficacy and safety across a broad body surface area, reflecting real-world use in a condition that often affects nearly the entire skin. Enrollment in both trials is expected to be completed in the first half of 2026, with top-line results anticipated in the second half of the year.

The US Food and Drug Administration has granted Orphan Drug Designation and Pediatric Rare Disease Designation for QRX003 in Netherton Syndrome, while the European Medicines Agency has also awarded Orphan Drug Designation.² Looking ahead, Quoin plans to submit a New Drug Application in the US and other territories in late 2026 or early 2027, contingent on positive results from the ongoing pivotal trials. If successful, QRX003 could mark a paradigm shift in the treatment of Netherton Syndrome, offering the first approved, targeted therapeutic option for a condition that has long lacked effective treatments.

References

1. Quoin Pharmaceuticals Files Breakthrough Medicine Designation Application in Saudi Arabia for QRX003 in Netherton Syndrome. News release. Globe Newswire. Published January 20, 2026. Accessed January 20, 2026. https://www.globenewswire.com/news-release/2026/01/20/3221722/0/en/Quoin-Pharmaceuticals-Files-Breakthrough-Medicine-Designation-Application-in-Saudi-Arabia-for-QRX003-in-Netherton-Syndrome.html

2. Quoin Pharmaceuticals Announces U.S. FDA Grants Orphan Drug Designation for QRX003 in Netherton Syndrome. News release. Quoin Pharmaceuticals. Published October 21, 2025. Accessed January 20, 2026. https://investors.quoinpharma.com/news-releases/news-release-details/quoin-pharmaceuticals-announces-us-fda-grants-orphan-drug