Quoin Pharmaceuticals Announces U.S. FDA Grants Orphan Drug Designation for QRX003 in Netherton Syndrome

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ: QNRX) announced the U.S. FDA has granted Orphan Drug Designation for QRX003 to treat Netherton Syndrome on Oct 21, 2025.

The FDA designation follows an EMA orphan designation in May 2025 and provides benefits including tax credits, fee waivers or reductions, and seven years of US market exclusivity if QRX003 is approved. QRX003 lotion (4%) is in two late‑stage pivotal whole‑body trials; enrollment is expected complete in Q1 2026, topline data is anticipated in H2 2026, and an NDA submission is planned later in 2026.

Positive

• FDA orphan designation granted on October 21, 2025
• EMA orphan designation previously granted in May 2025
• Seven years of potential US market exclusivity if approved
• Enrollment in two pivotal trials expected complete Q1 2026
• Top‑line pivotal trial data anticipated in H2 2026

Negative

• QRX003 remains investigational and is not yet approved
• Regulatory approval depends on pivotal trial results and NDA

Insights

Rare disease regulatory strategist

FDA orphan designation for QRX003 materially strengthens Quoin's regulatory advantages and supports a clearer approval pathway.

Quoin Pharmaceuticals gains U.S. Orphan Drug Designation for QRX003, which brings tangible regulatory benefits: eligibility for clinical testing tax credits, potential waiver or reduction of FDA application fees, and seven years of U.S. market exclusivity if approved.

The programmatic benefit reduces development costs and raises barriers to near‑term competition but does not guarantee approval. Key dependencies include completion of pivotal trial enrollment by Q1 2026, delivery of top‑line data in the second half of 2026, and a timely NDA submission later in that year.

Watch concrete milestones on the stated timeline: enrollment completion (Q1 2026), top‑line readout (second half of 2026), and NDA submission (later in 2026). These items will determine whether the regulatory advantages translate into commercial exclusivity.

Upon Approval of NDA Quoin Will Receive Seven Years Marketing Exclusivity for QRX003 in the US

Orphan Drug Designation previously granted by the European Medicines Agency in May 2025

ASHBURN, Va., Oct. 21, 2025 (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 Orphan Drug Designation (ODD) to its lead product candidate, QRX003, for the treatment of Netherton Syndrome. The designation follows previously granted Orphan Drug Designation by the European Medicines Agency (EMA) in May 2025.

The FDA's Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. QRX003 is on track to potentially become the first approved treatment for Netherton Syndrome.

“Receiving Orphan Drug Designation from the FDA is yet another important milestone in our mission to bring QRX003 to patients suffering with Netherton Syndrome,” said Dr. Michael Myers, CEO of Quoin Pharmaceuticals. “Together with the EMA designation granted earlier in the year, this latest recognition by the FDA could potentially help facilitate the pathway of QRX003 to approval in the US whilst providing significant data protection to the product, if approved. Quoin remains steadfastly committed to completing the clinical development of QRX003 with a high degree of urgency on behalf of patients and families living with this devastating disease.”

QRX003 lotion (4%) is being evaluated in two late-stage whole body pivotal clinical trials for Netherton Syndrome. Enrollment in both pivotal studies is expected to be completed in Q1 2026, top-line data is anticipated in the second half of 2026, and NDA submission is planned later in the year.

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 comprises several products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, Palmoplantar Keratoderma, Scleroderma, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma 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: QRX003 being on track to potentially become the first approved treatment for Netherton Syndrome, FDA recognition of Orphan Drug Designation potentially helping to facilitate the pathway of QRX003 to approval in the US whilst providing significant data protection, completing the clinical development of QRX003, completing enrollment in two late-stage whole body pivotal clinical trials for Netherton Syndrome, evaluating QRX003 lotion (4%) in two late-stage whole body pivotal clinical trials for Netherton Syndrome, completing enrollment in both pivotal studies in Q1 2026, receiving top-line data in the second half of 2026 and submitting an NDA later in the year, 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, Scleroderma, Epidermolysis Bullosa 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, 2024 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 did Quoin Pharmaceuticals (QNRX) announce on October 21, 2025 regarding QRX003?

The company announced the FDA granted Orphan Drug Designation to QRX003 for Netherton Syndrome on October 21, 2025.

What benefits does the FDA Orphan Drug Designation provide for QNRX's QRX003?

The designation provides benefits including tax credits, FDA fee waiver/partial payment, and seven years of US market exclusivity if approved.

What are the clinical timelines for QRX003 (QNRX) after the FDA designation?

Quoin expects pivotal trial enrollment complete in Q1 2026, top‑line data in H2 2026, and an NDA submission later in 2026.

Does FDA orphan designation mean QRX003 is approved for Netherton Syndrome?

No; orphan designation is a development incentive and does not equal approval; approval requires successful pivotal data and NDA review.

Was QRX003 previously recognized by European regulators before the FDA decision?

Yes; QRX003 received Orphan Drug Designation from the EMA in May 2025.