Vyjuvek

Vyjuvek is a prescription topical gel used to treat dystrophic epidermolysis bullosa (DEB). It is the first and only FDA-approved gene-therapy treatment for DEB that addresses the underlying cause and promotes wound healing in patients from birth through adulthood. Vyjuvek is applied weekly.

Key Facts About Vyjuvek

• FDA Approval: May 19, 2023 (Initial); Extended to newborns September 2025
• Age Range: Newborns and older (no minimum age)
• Condition Treated: Dystrophic epidermolysis bullosa (DEB) with COL7A1 gene mutations
• Generic Name: Beremagene-geperpavec-svdt
• Formulation: Topical gel
• Drug Class: HSV-1 vector-based gene therapy
• Administration: Weekly topical application
• Manufacturer: Krystal Biotech, Inc.

Dystrophic epidermolysis bullosa is a rare genetic skin disease that causes extremely fragile skin. Minor friction or trauma can cause severe blistering, wounds, and skin tears. DEB is caused by mutations in the COL7A1 gene, which produces collagen type VII protein essential for binding the skin's outer layer (epidermis) to the inner layer (dermis).

Vyjuvek treats the root cause of DEB through innovative gene therapy technology:

• Gene Delivery: Uses a modified, non-replicating herpes simplex virus type 1 (HSV-1) vector to safely deliver functional COL7A1 genes directly to DEB wounds
• Protein Production: The delivered genes enable skin cells to produce functional collagen type VII protein
• Wound Healing: New collagen helps reconnect the dermis and epidermis, promoting natural wound closure
• Non-Integrating: The therapy does not alter the patient's native genetic material.

This HSV-1 vector-based gene therapy represents a safe, non-invasive approach to treating DEB at the molecular level.

Vyjuvek is indicated for:

• Newborns, infants, children, and adults with dystrophic epidermolysis bullosa
• Patients confirmed to have mutation(s) in the COL7A1 gene
• Those with DEB-related wounds requiring treatment.

Updated September 2025: Vyjuvek is now approved for use in newborns, making it available to DEB patients from birth.

Common Side Effects (Incidence >5%)

• Itching
• Chills
• Redness
• Rash
• Cough
• A runny nose.

Important Safety Information

• No drug-related serious adverse events reported in clinical trials
• No treatment discontinuations due to adverse events in studies
• Pregnant individuals should not prepare, apply, or have direct contact with Vyjuvek-treated wounds.

Handle Vyjuvek with care to avoid accidental exposure. Avoid touching treated wounds and dressings for at least 24 hours after application. Properly dispose of used dressings and wash any accidental skin contact thoroughly.

Report adverse reactions: Contact Krystal Biotech at 1-844-557-9782 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

First-in-Class Gene Therapy

Vyjuvek remains the only FDA-approved treatment specifically indicated for dystrophic epidermolysis bullosa. While other wound care products exist (such as Filsuvez birch bark extract for EB), Vyjuvek is unique in treating the underlying genetic cause of DEB.

Comparison with Supportive Care Options

• Zevaskyn: Another topical treatment for epidermolysis bullosa
• Filsuvez: Birch bark extract gel for EB wound management
• Traditional wound care: Bandaging, infection prevention, pain management

Only Vyjuvek addresses the COL7A1 gene mutation directly through gene therapy.

Before your healthcare provider applies Vyjuvek, tell them about all your medical conditions, including any skin disorders you may have;

Individuals who are pregnant should not prepare or apply Vyjuvek gel and should avoid direct contact with the treated wounds or dressings from treated wounds.

Vyjuvek can be administered by a qualified healthcare provider or, after training, at home by a patient or caregiver.

• If Vyjuvek is applied by a healthcare provider, they will mix the biological suspension with the excipient gel immediately before use.
• If you or a caregiver is administering Vyjuvek at home, then syringes containing premixed Vyjuvek gel will be provided by the pharmacy in a sealable plastic bag. This gel is ready for administration. Vyjuvek gel syringes arrive refrigerated (2-8°C). Store refrigerated for up to 48 hours or at room temperature for up to 8 hours before use. Keep away from children. Never heat or microwave.

Applying Vyjuvek at Home

Products to Avoid Before Vyjuvek Treatment

Stop using these products on treatment areas approximately 24 hours before Vyjuvek application:

• CBD
• Calmoseptine
• Chlorhexidine
• Hibiclens
• Honey
• Hydrogen peroxide
• Octenisept
• Silver-containing products
• Tea tree oil
• Vashe
• Vinegar
• Zinc (concentrations above 12.8%).

These substances have antiviral properties that may reduce Vyjuvek's effectiveness. Contact your healthcare team if you have questions about other products.

If you've used any of these products, thoroughly rinse the wound with clean water or saline before Vyjuvek application. You may resume using these products after the first dressing change.

Application

• Apply the gel in evenly spaced droplets across the wound in a grid pattern (approximately 1cm x 1cm apart)
• Cover the treated wound with an appropriate wound dressing
• Apply once a week until the wound has healed.

Important Safety Instructions After Application

For the first 24 hours after treatment:

• Do not directly touch treated wounds or dressings
• Wear protective gloves when changing dressings
• Disinfect the first dressing change bandages with an anti-viral agent and seal in a plastic bag before disposal
• Seal subsequent dressings in plastic bags for household waste disposal
• Flush mucous membranes with clean water for 15 minutes if accidental exposure occurs.

For more information about using Vyjuvek, patients, caregivers, and healthcare professionals can call Krystal Connect at 1-844-5-KRYSTAL.

Age-Based Maximum Weekly Doses (Updated September 2025)

*Volume after mixing biological suspension with excipient gel PFU = plaque-forming units

Vyjuvek is applied to the skin only. The weekly dose that can be applied depends on the age of the patient.

• 6 months to <3 years: 1.6 x 109 PFU or 0.8mL of reconstituted gel
• 3 years and older: 3.2 x 109 PFU or 1.6mL of reconstituted gel.

Sometimes it is not safe to use certain medicines at the same time. Some drugs can affect your blood levels of other drugs you use, which may increase side effects or make the medicines less effective. Tell your doctor about all the other medications you apply to your skin or take by mouth.

No formal drug interaction studies have been performed with Vyjuvek.

See the Vyjuvek Package Insert for a full list of interactions.

Storage Conditions

• Primary storage: -15°C to -25°C (5°F to -13°F) in original carton
• Alternative refrigeration: 2°C to 8°C (35.6°F to 46.4°F) for up to 1 month.

After Mixing

• Room temperature: Use within 8 hours
• Refrigerated: Use within 48 hours
• No preservatives: Product contains no preservatives

Active Ingredient: Beremagene-geperpavec-svdt

• Modified HSV-1 vector-based gene therapy
• Replication-deficient and non-integrating
• Delivers 2 copies of the functional COL7A1 gene.

Formulation:

• Biological suspension: 1.0 mL at 5×10⁹ PFU/mL concentration
• Excipient gel: 1.5 mL fill volume
• Final mixed gel: 5.0×10⁹ PFU in 2.5 mL total volume