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Study Design

GEM-3, the phase 3 confirmatory study, was an intra-patient, randomized, double-blind, multicenter trial that evaluated the efficacy and safety of VYJUVEK1

In the trial, each patient was treated with both VYJUVEK and placebo1

  • Every patient had 2 cutaneous wounds similar in size, appearance, and anatomical region selected as the primary wound pair1,2
  • Each patient served as their own control, with one of their primary wounds treated with VYJUVEK and the other with placebo1,2

GEM-3 Study Design2,3

Tap the image to enlarge.

Chart of VYJUVEK™ GEM-3 study design
Chart of VYJUVEK™ GEM-3 study design

*Complete (100%) wound closure was defined as durable wound closure (skin re-epithelialization without drainage) evaluated at two consecutive visits two weeks apart.2

Patient characteristics2

Patient demographics/characteristics Total patients (N=31)
Age, years
Mean (SD) 17.2 (10.7)
Range 1-44
Age category, n (%)
≤12 years 10 (32.3)
>12 to ≤18 years 9 (29.0)
>18 years 12 (38.7)
Sex, n (%)
Male 20 (64.5)
Female 11 (35.5)
Race, n (%)
White 20 (64.5)
Asian 6 (19.4)
American Indian or Alaska Native 5 (16.1)
Genotype, n (%)
Dominant DEB (DDEB) 1 (3.2)
Recessive DEB (RDEB) 30 (96.8)

GEM-3 patient eligibility criteria3

Inclusion Criteria

  • Informed consent or assent form received
  • Age >6 months with genetically confirmed DEB diagnosis
  • Two cutaneous wounds meeting selection criteria:
    • Similar size, anatomical region, appearance
    • Clean, adequate granulation tissue, excellent vascularization, not infected
  • Subjects and caregivers are able to understand and cooperate with the study procedures, and will attend all required follow-up visits
  • Male, or female of childbearing potential, must use a reliable birth control method throughout the study and for three (3) months post-last dose of VYJUVEK
  • Negative pregnancy test at Visit 1 (Week 1), if applicable

Exclusion Criteria

  • Medical instability limiting travel
  • Diseases or conditions that could interfere with compliance or study assessments
  • Current evidence or a history of squamous cell carcinoma in the treatment area
  • Subjects actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1)
  • Active drug or alcohol addiction
  • Hypersensitivity to local anesthesia (lidocaine/prilocaine cream)
  • Participation in an interventional clinical trial within the past three (3) months (not including VYJUVEK administration)
  • Receipt of a skin graft in the past three (3) months
  • Pregnant or nursing women

Learn about VYJUVEK efficacy and safety

View Efficacy & Safety Data
INDICATION AND USAGE

VYJUVEK is a topical gel indicated for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII (COL7A1) gene.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Accidental Exposure to VYJUVEK gel: VYJUVEK will not replicate in the subject’s cells and does not integrate into the subject cells’ native genetic material. For precautions, avoid direct contact with treated wounds (e.g., touching or scratching) and dressings of treated wounds for approximately 24 hours following treatment. Wear protective gloves when assisting subjects with changing wound dressings and handling the disposal. In the event of accidental exposure (e.g., through a splash to the eyes or mucous membranes), flush with clean water for at least 15 minutes.

Clean all surfaces that may have come in contact with VYJUVEK biological suspension or gel and treat all spills with a virucidal agent.

Dispose all materials (e.g., vial, syringe, needle, cleaning materials) that may have come in contact with VYJUVEK biological suspension or gel into a biohazard bag or container.

ADVERSE REACTIONS

The most common adverse reactions (>5%) were itching, chills, redness, rash, cough, and runny nose.


References

1. VYJUVEK [prescribing information]. Krystal Biotech, Inc.; 2023.

2. Marinkovich MP, Gonzalez ME, Guide S, et al. GEM-3: a phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). Presented at 2022 AAD Annual Meeting; March 27, 2022; Boston, MA.

3. Krystal Biotech, Inc. A phase III double blinded, placebo-controlled, efficacy and safety study of beremagene geperpavec (B-VEC, previously “KB103”) for the treatment of dystrophic epidermolyis bullosa (DEB). ClinicalTrials.gov identifier: NCT04491604. Updated August 3, 2022. Accessed January 10, 2023. https://clinicaltrials.gov/ct2/show/record/NCT04491604

IMPORTANT SAFETY INFORMATION
INDICATION AND USAGE

VYJUVEK is a topical gel indicated for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII (COL7A1) gene.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Accidental Exposure to VYJUVEK gel: VYJUVEK will not replicate in the subject’s cells and does not integrate into the subject cells’ native genetic material. For precautions, avoid direct contact with treated wounds (e.g., touching or scratching) and dressings of treated wounds for approximately 24 hours following treatment. Wear protective gloves when assisting subjects with changing wound dressings and handling the disposal. In the event of accidental exposure (e.g., through a splash to the eyes or mucous membranes), flush with clean water for at least 15 minutes.

Clean all surfaces that may have come in contact with VYJUVEK biological suspension or gel and treat all spills with a virucidal agent.

Dispose all materials (e.g., vial, syringe, needle, cleaning materials) that may have come in contact with VYJUVEK biological suspension or gel into a biohazard bag or container.

ADVERSE REACTIONS

The most common adverse reactions (>5%) were itching, chills, redness, rash, cough, and runny nose.


References

1. VYJUVEK [prescribing information]. Krystal Biotech, Inc.; 2023.

2. Marinkovich MP, Gonzalez ME, Guide S, et al. GEM-3: a phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). Presented at 2022 AAD Annual Meeting; March 27, 2022; Boston, MA.

3. Krystal Biotech, Inc. A phase III double blinded, placebo-controlled, efficacy and safety study of beremagene geperpavec (B-VEC, previously “KB103”) for the treatment of dystrophic epidermolyis bullosa (DEB). ClinicalTrials.gov identifier: NCT04491604. Updated August 3, 2022. Accessed January 10, 2023. https://clinicaltrials.gov/ct2/show/record/NCT04491604