Krystal Biotech Announces FDA Approval of Updated VYJUVEK® Label

Rhea-AI Summary

Krystal Biotech (NASDAQ: KRYS) has received FDA approval for important updates to the VYJUVEK® label, its treatment for dystrophic epidermolysis bullosa (DEB). The key changes include expanding patient eligibility to include newborns and allowing patients and caregivers to apply the treatment themselves.

The updated label also provides greater flexibility in wound dressing management, allowing dressing removal during the next change rather than waiting 24 hours. These modifications are supported by real-world data and results from an open-label extension study, demonstrating VYJUVEK's long-term safety and efficacy across all age groups.

Insights

Biotech Regulatory Analyst

FDA label expansion for VYJUVEK increases accessibility and convenience, strengthening Krystal Biotech's market position in DEB treatment.

The FDA's approval of Krystal Biotech's updated label for VYJUVEK represents a significant enhancement to the product's market positioning and accessibility. The label modification delivers two key improvements: expansion of the eligible patient population to include DEB patients from birth (previously age-restricted), and authorization for self-application by patients and caregivers rather than requiring healthcare professional administration.

These changes substantively improve the product's commercial profile by removing important barriers to adoption. The age expansion increases the addressable patient population, while the self-application allowance dramatically improves convenience and reduces the logistical and financial burdens associated with healthcare professional administration. Additionally, the new flexibility regarding wound dressing management (allowing removal during next dressing change rather than waiting 24 hours) better integrates VYJUVEK into existing care routines.

The label update is supported by real-world evidence and extension study data, indicating the FDA is satisfied with VYJUVEK's safety profile even with expanded usage parameters. For rare disease therapies like VYJUVEK, such label expansions are particularly valuable as they maximize utility within an already limited patient population. The endorsement from debra of America (a patient advocacy organization) further validates the meaningful impact these changes will have on patient quality of life.

This regulatory win strengthens VYJUVEK's position as the standard of care for DEB wound treatment across all severity levels and should positively impact compliance rates – a critical factor for both patient outcomes and consistent revenue generation for Krystal Biotech.

Revised label allows treatment of DEB patients from birth

VYJUVEK can now be applied by patients and caregivers

PITTSBURGH, Sept. 15, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the United States Food and Drug Administration (FDA) approved a label update for VYJUVEK^® (beremagene geperpavec-svdt) that expands the VYJUVEK eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and provides patients full flexibility with respect to VYJUVEK application and managing wound dressings.

“We believe these changes further reinforce VYJUVEK’s leadership position as the most flexible, convenient, and disease modifying medicine to treat DEB patients in the United States,” said Krish S. Krishnan, Chairman and CEO of Krystal Biotech. “Moreover, by providing patients and their caregivers the ability to apply VYJUVEK themselves, we have made it easier for patients to integrate VYJUVEK into their daily routines and lifestyle. Overall, we believe that these advancements solidify VYJUVEK as the standard of care for all DEB wounds, regardless of wound size and severity, and will further improve compliance.”

In addition to allowing DEB patients and their caregivers to apply VYJUVEK at home on their own, today’s label update also affords patients greater flexibility in managing wound dressings. Wound dressings are now permitted to be removed as part of the next dressing change rather than waiting 24 hours, further integrating VYJUVEK into existing wound care routines.

“The updates to the VYJUVEK label are yet another significant and impactful step forward for all those living with DEB” said Brett Kopelan, Executive Director of debra of America. “Enabling caretakers to apply VYJUVEK during their standard of care regimen is an enormous positive change allowing for increased convenience without sacrificing safety. The Krystal team has always prioritized patient safety and convenience when it comes to the use of VYJUVEK and them advocating for these updates is not surprising given Krystal’s patient centric approach. This aspect of the update to the label will only increase the quality of life of those living with this challenging disorder and that is exactly what our community needs.”

This label update is based on real-world data collected since VYJUVEK launch in the United States, as well as results from the open label extension study conducted in the United States and published earlier this year, which collectively reinforce the long-term safety and efficacy of VYJUVEK across patients of all ages, including in cases of patient or caregiver application.

About VYJUVEK
VYJUVEK is a non-invasive, topical, redosable genetic medicine designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. VYJUVEK was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. VYJUVEK is approved in the United States, Europe, and Japan.

U.S. INDICATION
VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

IMPORTANT SAFETY INFORMATION

Adverse Reactions
The most common adverse drug reactions (incidence >5%) were itching, chills, redness, rash, cough, and runny nose. These are not all the possible side effects with VYJUVEK. Call your healthcare provider for medical advice about side effects.

To report SUSPECTED ADVERSE REACTIONS, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Contraindications
None

Warnings and Precautions
VYJUVEK gel may be applied by a healthcare provider, a caregiver, or the patient.

After treatment, patients and caregivers should be careful not to touch treated wounds and dressings until the next dressing change.

Wash hands and wear protective gloves when changing wound dressings. Disinfect bandages from the first dressing change with a virucidal agent and dispose of the disinfected bandages in a separate sealed plastic bag in household waste. Dispose of the subsequent used dressings in a sealed plastic bag in household waste.

Patients should avoid touching or scratching wound sites or wound dressings.

In the event of an accidental exposure flush with clean water for at least 15 minutes.

For more information, see full U.S. Prescribing Information.

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 in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter).

Forward-Looking Statements

This press release contains “forward looking” statements within the meaning of the Private Securities Litigation Reform Act of 1995 based on the Company’s current expectations and beliefs regarding the recent FDA approved label update for VYJUVEK. All statements other than historical facts are or may be deemed to be forward-looking statements and involve known and unknown risks, uncertainties, and assumptions that could cause actual results to differ materially from those indicated by such forward-looking statements as a result of various important factors set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. The Company provides this information as of the date of this release and assumes no obligation to update any forward-looking statements.

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

FAQ

What are the new changes to Krystal Biotech's VYJUVEK label approved by the FDA?

The FDA approved two major changes: extending VYJUVEK treatment eligibility to DEB patients from birth and allowing patients and caregivers to apply the treatment themselves. Additionally, wound dressings can now be removed during the next dressing change rather than waiting 24 hours.

How does the new VYJUVEK label update benefit DEB patients?

The update allows treatment from birth, enables self-administration by patients and caregivers, and provides more flexible wound dressing management, making the treatment more convenient and accessible while maintaining safety.

What evidence supports the new VYJUVEK label changes by Krystal Biotech?

The label updates are supported by real-world data collected since VYJUVEK's launch and results from an open-label extension study in the United States, both confirming long-term safety and efficacy across all age groups.

Can VYJUVEK now be applied at home without healthcare professional supervision?

Yes, the new label update allows patients and caregivers to apply VYJUVEK at home on their own, making it easier to integrate the treatment into daily routines.

What is the recommended timing for VYJUVEK wound dressing changes under the new label?

Under the new label, wound dressings can be removed during the next dressing change, eliminating the previous 24-hour waiting requirement and better aligning with existing wound care routines.