Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome

Quoin Pharmaceuticals (NASDAQ:QNRX) announced that Japan's Ministry of Health, Labour and Welfare granted Orphan Drug Designation to QRX003 for Netherton Syndrome, a rare genetic skin disorder with no approved treatments.

Rhea-AI Summary

Quoin Pharmaceuticals (NASDAQ:QNRX) announced that Japan's Ministry of Health, Labour and Welfare granted Orphan Drug Designation to QRX003 for Netherton Syndrome, a rare genetic skin disorder with no approved treatments. The status provides development incentives and up to 10 years of market exclusivity upon approval.

QRX003, a 4% lotion, is in Phase 2 whole-body trials, with a pivotal Phase 3 study expected in the second half of 2026 and a potential NDA filing in 2027. QRX003 already holds U.S. FDA Orphan, Pediatric Rare Disease and Fast Track designations, plus EMA Orphan status.

Positive

• Japan MHLW Orphan Drug Designation for QRX003 in Netherton Syndrome
• Up to 10 years of market exclusivity in Japan upon approval
• Multiple global regulatory designations from FDA and EMA for QRX003
• QRX003 currently in Phase 2 whole-body clinical trials
• Pivotal Phase 3 study planned for second half of 2026
• Potential NDA filing for QRX003 targeted for 2027

Negative

• QRX003 still in clinical development with no approvals yet
• Pivotal Phase 3 trial has not yet started as of June 2026

Argus Jun 4 session

-3.16% close to close Open Argus

Details

News Market Reaction – QNRX

On Jun 4, the day this news came out, QNRX closed 3.16% below the previous close.

Data tracked by StockTitan Argus for the Jun 4 session.

Key Figures

Japan prevalence threshold: Fewer than 50,000 patients Japan exclusivity: Up to 10 years QRX003 concentration: 4% +3 more

Japan prevalence threshold

Fewer than 50,000 patients

Definition of rare disease for MHLW Orphan Drug Designation

Japan exclusivity

Up to 10 years

Market exclusivity in Japan upon approval under Orphan Drug Designation

QRX003 concentration

4%

QRX003 lotion strength used in ongoing trials

Phase 2 status

Phase 2

Whole-body clinical trials in Netherton Syndrome underway

Phase 3 timing

Second half of 2026

Expected initiation of pivotal Phase 3 study for QRX003

NDA timing goal

2027

Potential NDA filing year for QRX003 in Netherton Syndrome

Previous Clinical trial Reports

5 past events · Latest: 2026-06-02

Same Type 5 events

1. 2026-06-02

   IND for PSS

   24h Move

   -4.4%

   IND filing for QRX003 in Peeling Skin Syndrome backed by 15 months use.

2. 2026-01-27

   Japan ODD submission

   24h Move

   -1.7%

   Submission to MHLW for Orphan and Fast Track status for QRX003 in Japan.

3. 2025-05-20

   EMA ODD granted

   24h Move

   +6.0%

   EMA Orphan Drug Designation for QRX003 with EU exclusivity incentives.

4. 2025-05-14

   Positive PSS data

   24h Move

   +20.3%

   Initial positive clinical data for QRX003 in pediatric Peeling Skin Syndrome.

5. 2025-04-02

   Positive NS data

   24h Move

   +38.2%

   Additional whole-body QRX003 data in pediatric Netherton Syndrome with no AEs.

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

Key Terms

orphan drug designation, pediatric rare disease designation, fast track designation, ministry of health, labour and welfare (mhlw), +4 more

8 terms

orphan drug designation regulatory

"has granted Orphan Drug Designation to QRX003 for the treatment of Netherton Syndrome"

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

pediatric rare disease designation regulatory

"Follows Orphan Drug Designation, Pediatric Rare Disease Designation, and Fast Track Designation previously granted"

A pediatric rare disease designation is a formal regulatory status given to a drug or therapy aimed at a serious, uncommon illness that primarily affects children. It matters to investors because the designation often brings financial and timing advantages—such as development funding, tax breaks, faster review, or market protections—similar to getting a VIP pass that lowers costs and increases the chance of reaching patients and generating returns.

fast track designation regulatory

"Pediatric Rare Disease Designation, and Fast Track Designation previously granted by the U.S. FDA"

Fast track designation is a status the U.S. Food and Drug Administration grants to drugs intended to treat serious conditions and address an unmet medical need. It gives the developer more frequent communication with the FDA and can allow parts of the application to be reviewed on a rolling basis, and it may pave the way to priority review or accelerated approval. It can shorten development timelines, though it does not guarantee approval.

ministry of health, labour and welfare (mhlw) regulatory

"Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation"

Japan’s Ministry of Health, Labour and Welfare is the national government agency responsible for public health, medical regulations, labor standards, and social welfare programs. Investors care because its approvals, safety rules, and policy decisions can change market access, costs, and demand for healthcare, pharmaceutical, and labor-intensive businesses—think of it as the rule-maker and safety inspector whose decisions can speed, delay, or reshape a company’s ability to sell products and hire workers.

netherton syndrome medical

"for the treatment of Netherton Syndrome, a rare and severe genetic skin disorder"

A rare inherited skin and immune disorder that causes fragile, scaly, inflamed skin, frequent infections, and fragile or unusual hair, like a house with faulty waterproofing that lets problems in. For investors, it matters because the small patient population, severe unmet medical need, and predictable biological cause can make treatments eligible for special regulatory incentives, faster development paths, and premium pricing if a safe, effective therapy is approved.

breakthrough medicine designation regulatory

"Quoin has also filed an application for Breakthrough Medicine Designation with the Saudi Food and Drug Authority"

A breakthrough medicine designation is a formal status granted by a drug regulator that signals a new treatment shows substantial promise for a serious condition and qualifies for faster, more intensive review. For investors it matters because the designation can shorten development time, reduce some regulatory hurdles and raise the likelihood of eventual approval, similar to giving a promising product a VIP fast‑track through the approval process which can boost a company’s value and investor interest.

phase 2 medical

"QRX003 lotion (4%) is currently being evaluated in Phase 2 whole-body clinical trials"

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

phase 3 medical

"Quoin's pivotal Phase 3 study is expected to initiate in the second half of 2026"

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

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-Designation provides regulatory and development incentives in Japan, including up to 10 years of market exclusivity upon approval-

-Follows Orphan Drug Designation, Pediatric Rare Disease Designation, and Fast Track Designation previously granted by the U.S. FDA, and Orphan Drug Designation previously granted by the European Medicines Agency-

-Quoin is working closely with leading Japanese clinicians to refine clinical and regulatory pathway for approval-

ASHBURN, Va., June 04, 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 Japan's Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug Designation to QRX003 for the treatment of Netherton Syndrome, a rare and severe genetic skin disorder for which there are currently no approved treatments.

The MHLW grants Orphan Drug Designation to medicines intended to treat rare diseases that affect fewer than 50,000 patients in Japan and for which there is high unmet medical need. The designation provides certain development incentives, including prioritized consultation, reduced consultation and application fees, tax incentives, priority review of applications, and up to 10 years of market exclusivity upon approval.

The MHLW designation adds to the global regulatory recognition QRX003 has received for Netherton Syndrome, which includes Orphan Drug Designation, Pediatric Rare Disease Designation, and Fast Track Designation from the U.S. Food and Drug Administration, and Orphan Drug Designation from the European Medicines Agency. Quoin has also filed an application for Breakthrough Medicine Designation with the Saudi Food and Drug Authority.

"Receiving Orphan Drug Designation from Japan's MHLW adds an additional important regulatory milestone for QRX003 and its potential as a safe and effective treatment for Netherton Syndrome," said Dr. Michael Myers, CEO of Quoin Pharmaceuticals. "Japan is a strategically important market for QRX003, and, along with the US and Western Europe, is one of three core territories in which we plan to self-commercialize QRX003 and our other pipeline products, once approved. This designation is another important step in that strategy and complements the regulatory recognition QRX003 has received in the United States and Europe."

QRX003 lotion (4%) is currently being evaluated in Phase 2 whole-body clinical trials in patients with Netherton Syndrome. Quoin's pivotal Phase 3 study is expected to initiate in the second half of 2026, with potential NDA filing in 2027. Quoin is working closely with leading Japanese clinicians to refine the clinical and regulatory pathway for approval of QRX003 for the treatment of Netherton Syndrome.

About Netherton Syndrome

Netherton Syndrome is a rare, inherited skin disorder caused by mutations in the SPINK5 gene, leading to severe skin barrier dysfunction, chronic inflammation, and a heightened risk of infections and allergic complications. Patients often experience widespread skin redness, scaling, persistent itching, and significant impairment in quality of life. There are currently no FDA-approved therapies for the treatment of Netherton Syndrome, and treatment options are limited to supportive care and off-label therapies.

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: QRX003 ‘s potential as a safe and effective treatment for Netherton Syndrome; the strategic importance of Japan as a market for QRX003; plans to self-commercialize QRX003 and Quoin’s other pipeline products in Japan, the US and Western Europe, once approved; initiating Quoin's pivotal Phase 3 study in the second half of 2026, with potential NDA filing in 2027; working closely with leading Japanese clinicians to refine clinical and regulatory pathway for approval of QRX003 for the treatment of Netherton Syndrome;; 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, 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

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did Quoin Pharmaceuticals (QNRX) announce about QRX003 in Japan on June 4, 2026?

Quoin announced that Japan's MHLW granted Orphan Drug Designation to QRX003 for Netherton Syndrome. According to Quoin, this status for the rare skin disorder brings development incentives, priority review and potential long-term market exclusivity in Japan upon regulatory approval.

What benefits does Japan's Orphan Drug Designation give QRX003 and QNRX shareholders?

The designation provides prioritized consultation, reduced fees, tax incentives, priority review and up to 10 years of market exclusivity upon approval. According to Quoin, these benefits could support QRX003's development economics and potential future commercial position in the Japanese Netherton Syndrome market.

What other regulatory designations does QRX003 have besides Japan's MHLW Orphan status?

QRX003 has Orphan Drug, Pediatric Rare Disease and Fast Track designations from the U.S. FDA and Orphan Drug Designation from the European Medicines Agency. According to Quoin, it has also filed for Breakthrough Medicine Designation with the Saudi Food and Drug Authority.

What is the clinical development stage of QRX003 for Netherton Syndrome as of 2026?

QRX003 lotion (4%) is being evaluated in Phase 2 whole-body clinical trials in Netherton Syndrome patients. According to Quoin, a pivotal Phase 3 study is expected to start in the second half of 2026, supporting a potential regulatory submission thereafter.

When could Quoin Pharmaceuticals (QNRX) file an NDA for QRX003 in Netherton Syndrome?

Quoin expects a potential New Drug Application (NDA) filing in 2027 for QRX003. According to Quoin, this timeline depends on results from the ongoing Phase 2 studies and the planned pivotal Phase 3 trial in Netherton Syndrome patients.

How important is the Japanese market for QRX003 commercialization by Quoin Pharmaceuticals?

Quoin views Japan as a strategically important core territory alongside the US and Western Europe. According to Quoin, the company plans to self-commercialize QRX003 and other pipeline products in these regions, subject to achieving regulatory approvals.

How is Quoin Pharmaceuticals collaborating in Japan to advance QRX003 approval?

Quoin is working closely with leading Japanese clinicians to refine the clinical and regulatory pathway for QRX003. According to Quoin, this collaboration aims to support future approval of QRX003 for treating Netherton Syndrome in the Japanese market.