Krystal Biotech Announces United Kingdom MHRA Approval of VYJUVEK® for the Treatment of Dystrophic Epidermolysis Bullosa

Rhea-AI Summary

Krystal Biotech (NASDAQ:KRYS) received United Kingdom MHRA marketing authorization for VYJUVEK to treat wounds in patients with dystrophic epidermolysis bullosa (DEB) with COL7A1 mutations, from birth. VYJUVEK is the first genetic medicine approved in the UK for DEB.

The approval includes flexible administration at home or in healthcare settings, including by patients or caregivers. VYJUVEK met UK Orphan Designation criteria, enabling up to 12 years of market exclusivity. Launch timing will depend on completion of reimbursement procedures.

Positive

• MHRA approval of VYJUVEK for DEB with COL7A1 mutations from birth
• First genetic medicine approved in the UK for treatment of DEB
• Flexible dosing options including at-home administration and caregiver application
• UK Orphan Designation provides up to 12 years of market exclusivity
• Approval supported by Phase 1/2 GEM-1, Phase 3 GEM-3, extension, and real-world data
• Fourth major regulatory approval following United States, European Union, and Japan

Negative

• United Kingdom launch timing depends on completion of reimbursement procedures

News Market Reaction – KRYS

-4.75%

-4.75% Session close to close

In the May 18 session, KRYS declined 4.75%, reflecting a moderate negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement adds UK MHRA marketing authorization and up to 12 years of market exclusivity for ...

Analysis

This announcement adds UK MHRA marketing authorization and up to 12 years of market exclusivity for VYJUVEK, building on prior approvals in the US, EU, and Japan. The decision relied on Phase 1/2 GEM-1 and Phase 3 GEM-3 data plus an open-label extension, reinforcing the clinical foundation. Investors may track progress on UK reimbursement procedures, uptake under flexible at-home administration, and how this complements previously reported revenue growth and profitability from VYJUVEK.

Key Figures

MHRA approval date: May 15, 2026 Market exclusivity: up to 12 years Phase 1/2 study: GEM-1 +5 more

8 metrics

MHRA approval date May 15, 2026 UK MHRA marketing authorization for VYJUVEK

Market exclusivity up to 12 years UK Orphan Designation market exclusivity for VYJUVEK

Phase 1/2 study GEM-1 Part of clinical dataset supporting MHRA approval

Phase 3 study GEM-3 Pivotal trial supporting VYJUVEK MHRA approval

US approval date May 2023 FDA approval of VYJUVEK in the United States

EU approval date April 2025 European Commission approval of VYJUVEK

Japan approval date July 2025 Japan MHLW approval of VYJUVEK

Gene target COL7A1 Mutated gene addressed by VYJUVEK in DEB patients

Historical Context

5 past events · Latest: May 07 (Neutral)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
May 07	Investor conference	Neutral	+2.7%	Participation in BofA 2026 Health Care Conference with webcast access.
May 04	Earnings report	Positive	+7.9%	Strong Q1 2026 results with VYJUVEK revenue and solid cash balance.
Apr 30	Scientific conferences	Neutral	+0.9%	Multiple May–June presentations on gene therapy and oncology programs.
Apr 21	Earnings date notice	Neutral	-1.4%	Announcement of Q1 2026 earnings release and conference call timing.
Feb 24	Investor conference	Neutral	-2.8%	TD Cowen health care conference fireside chat and investor meetings.

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

Pattern Detected

Recent news, especially earnings and conference items, has generally seen aligned price reactions, with positive financial updates drawing stronger gains.

Recent Company History

Over the last six months, KRYS news has centered on investor outreach and financial strength. A BofA health care conference appearance on May 7, 2026 and other conference presentations in April and February saw modest price moves. The May 4, 2026 first-quarter results highlighted strong VYJUVEK revenue and were followed by a 7.86% gain. Today’s UK MHRA approval adds another commercial/regulatory milestone on top of this trajectory.

Key Terms

mhra, marketing authorization, orphan designation, orphan register, +4 more

8 terms

mhra regulatory

"the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization"

The MHRA is the United Kingdom’s government agency that checks and approves medicines, medical devices and vaccines before they can be sold, and monitors their safety once on the market. For investors, MHRA decisions act like a building inspector’s sign-off or a traffic controller’s clearance—approval clears the way for sales and revenue, while safety warnings, recalls or delays can slow launches, raise costs or hurt a product’s commercial prospects.

marketing authorization regulatory

"MHRA granted marketing authorization to VYJUVEK"

An official government approval that allows a drug, vaccine, or medical device to be sold and promoted in a specific country or region. Think of it as a safety and effectiveness passport issued after regulators review the product’s tests and manufacturing; for investors, receiving this authorization typically unlocks sales, revenue potential, and lower regulatory risk, while delays or denials can substantially affect a company’s value and timeline.

orphan designation regulatory

"VYJUVEK also fulfilled the United Kingdom Orphan Designation criteria"

Orphan designation is a regulatory label granted to a drug or therapy intended to treat a rare disease, which typically affects a small number of patients. It matters to investors because it unlocks incentives—such as development cost reductions, tax credits, priority review and a period of market exclusivity—that improve the commercial and regulatory prospects of a product; think of it as a government-backed head start and safety net for a niche but potentially high-value medicine.

orphan register regulatory

"will be added to the Orphan Register held by the MHRA"

An orphan register is a secure list or database of patients who have a rare disease, created to collect information about diagnosis, treatment and outcomes over time. Investors should care because these registries help developers prove a medicine works and is safe, speed up regulatory approval or reimbursement, and show the size and needs of a niche market—think of it like a detailed contact and history book for a small, hard-to-reach customer group.

phase 1/2 medical

"results from the Company’s Phase 1/2 GEM-1 and Phase 3 GEM-3 studies"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

phase 3 medical

"Phase 1/2 GEM-1 and Phase 3 GEM-3 studies"

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.

open label extension medical

"further supported by results from the Company’s open label extension study"

An open-label extension is a follow-on phase of a clinical trial where participants keep receiving the experimental drug and both doctors and patients know what treatment is being given. It matters to investors because it produces longer-term safety and effectiveness information, helps regulators and companies assess ongoing benefits or risks, and can indicate whether a therapy has staying commercial value — like an extended test drive revealing durability and real-world performance.

market exclusivity regulatory

"allowing it to benefit from up to 12 years of market exclusivity"

Market exclusivity is a limited legal protection that prevents rivals from selling the same drug or product for a set time, even if others could otherwise make a copy. It’s like a temporary shop window reserved for one seller, giving that company sole access to customers for that product. For investors, exclusivity can mean predictable sales and higher profit margins during the protected period, and the impending end of exclusivity is a key risk factor.

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

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VYJUVEK approved for the treatment of DEB from birth with flexible administration options similar to those already granted in the United States, European Union, and Japan 

VYJUVEK is the first genetic medicine approved in the United Kingdom for the treatment of DEB

PITTSBURGH, May 18, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced that, on May 15, 2026, the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization to VYJUVEK^® (beremagene geperpavec-svdt) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth.

VYJUVEK is the first genetic medicine approved in the United Kingdom for the treatment of DEB and is designed to address the root cause of the disease by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional type VII collagen protein expression with redosing. The approval in the United Kingdom also includes flexible administration options similar to those granted in the United States, European Union, and Japan, allowing for dosing at home or in a healthcare setting, with the option for administration by patients or their caregivers.

“This latest approval brings VYJUVEK closer to patients in the United Kingdom and advances our vision of providing corrective therapy to as many DEB patients as possible,” said Laurent Goux, Executive Vice President, Head of International at Krystal Biotech. “We are now focused on close collaboration with relevant authorities to support broad and rapid access across the United Kingdom.”

VYJUVEK also fulfilled the United Kingdom Orphan Designation criteria and will be added to the Orphan Register held by the MHRA, allowing it to benefit from up to 12 years of market exclusivity.

The approval of VYJUVEK by the MHRA was based on a comprehensive clinical dataset including results from the Company’s Phase 1/2 GEM-1 and Phase 3 GEM-3 studies, which collectively provided clear clinical evidence of successful COL7A1 gene delivery and durable wound closure following topical administration, and was further supported by results from the Company’s open label extension study and real-world experience with VYJUVEK.

The timing for launch of VYJUVEK in the United Kingdom will depend on completion of reimbursement procedures currently underway.

“We are delighted that VYJUVEK is now approved in the United Kingdom as the first and only corrective therapy for the treatment of DEB,” said Suma Krishnan, President of Research and Development at Krystal Biotech. “This positive decision by the MHRA, our fourth approval following the United States, European Union, and Japan, reflects both the strength of our clinical dataset and our commitment to maximizing access to VYJUVEK worldwide.”

VYJUVEK was previously approved by the Food and Drug Administration in the United States in May 2023, the European Commission in April 2025, and Japan’s Ministry of Health, Labour and Welfare in July 2025.

UNITED KINGDOM INDICATION
VYJUVEK is indicated for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth.

About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK^®, the Company’s first commercial product, is the first-ever redosable gene therapy and the first genetic medicine approved in the United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. Visit www.krystalbio.com to learn more or follow us on LinkedIn and X.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements regarding the timing of the launch of, and access to, VYJUVEK in the United Kingdom, and the completion of related reimbursement procedures; the potential duration of orphan market exclusivity in the United Kingdom; and the Company’s commitment to maximizing access to VYJUVEK worldwide. Words such as “anticipate,” “believe,” “expect,” “intend,” “plan,” “will,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on the Company’s current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including risks related to reimbursement for VYJUVEK; uncertainties associated with the maintenance of orphan designation and exclusivity in the United Kingdom; commercial and competitive risks; and such other important factors as are set forth under the caption “Risk Factors” in the Company’s most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q filed with the U.S. Securities and Exchange Commission. The Company undertakes no obligation to update any forward-looking statements except as required by law.

CONTACT
Investors and Media:                                                             
Stéphane Paquette, PhD
Krystal Biotech
spaquette@krystalbio.com                                    

FAQ

What did the MHRA approve for Krystal Biotech (NASDAQ:KRYS) on VYJUVEK in May 2026?

The MHRA granted marketing authorization for VYJUVEK to treat wounds in dystrophic epidermolysis bullosa (DEB) patients with COL7A1 mutations from birth. According to Krystal Biotech, this is the first genetic medicine approved in the United Kingdom for treatment of DEB.

Who can receive VYJUVEK treatment for DEB in the United Kingdom?

VYJUVEK is indicated for treating wounds in DEB patients with mutation(s) in the COL7A1 gene, from birth. According to Krystal Biotech, the authorization covers all ages with qualifying COL7A1 mutations, making it available to both pediatric and adult DEB patients in the UK.

What flexible administration options does VYJUVEK offer DEB patients in the UK?

VYJUVEK can be administered at home or in a healthcare setting, with application by patients or caregivers. According to Krystal Biotech, these options mirror those in the United States, European Union, and Japan, potentially easing access and integrating treatment into daily care routines.

What clinical data supported the MHRA approval of VYJUVEK for DEB?

The MHRA decision was based on Phase 1/2 GEM-1 and Phase 3 GEM-3 studies showing COL7A1 gene delivery and durable wound closure. According to Krystal Biotech, additional support came from an open-label extension study and real-world experience with topical VYJUVEK administration.

What does UK Orphan Designation mean for VYJUVEK and Krystal Biotech shareholders?

UK Orphan Designation allows VYJUVEK to benefit from up to 12 years of market exclusivity. According to Krystal Biotech, VYJUVEK will be added to the MHRA Orphan Register, which may help protect its DEB position during that exclusivity period in the United Kingdom.

When will VYJUVEK launch in the United Kingdom for DEB patients?

VYJUVEK’s UK launch timing will depend on completing reimbursement procedures currently underway. According to Krystal Biotech, the company is focusing on working with relevant authorities to support broad and rapid access, but no specific commercial availability date has been provided yet.

In which other regions is VYJUVEK already approved before the UK MHRA decision?

VYJUVEK was previously approved in the United States in May 2023, the European Union in April 2025, and Japan in July 2025. According to Krystal Biotech, the MHRA decision represents the fourth major regulatory approval for VYJUVEK worldwide.