Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa
- Dystrophic Epidermolysis Bullosa (DEB) is a serious rare genetic disease that affects the skin and mucosal tissues and is caused by one or more mutations in the COL7A1 gene, resulting in lack of production of functional type VII collagen (COL7) protein
- VYJUVEK is a topical gel that addresses the genetic cause of DEB by restoring functional copies of the COL7A1 gene to patients and is the only medicine available for patients in the US
- VYJUVEK is approved for the treatment of patients six months of age or older with either recessive or dominant DEB
- VYJUVEK is approved to be administered by a healthcare professional either in a healthcare professional setting (e.g., clinic) or a home setting
- Data from the pivotal Phase 3 (GEM-3) trial, published in the
New England Journal of Medicine, demonstrated that the trial met its primary endpoint of complete wound healing at six months and its key secondary endpoint of complete wound healing at three months
- Rare Pediatric Disease Priority Review Voucher granted
- Company to host investor conference call on
Friday, May 19, 2023at 6:00 pm ET
DEB is a rare and serious disease that affects the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene. The COL7A1 gene is responsible for the production of functional COL7 protein that forms anchoring fibrils necessary to bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears with minor friction or trauma. DEB patients suffer from open wounds, which lead to recurrent skin infections and fibrosis that can cause fusion of fingers and toes, and ultimately increase the risk of developing an aggressive form of skin cancer.
“This is a devastating disease,” said
The FDA approval of VYJUVEK is based on two clinical studies. The GEM-1/2 trial was an intra-patient, open label, single center, randomized, placebo-controlled study showing that repeat topical applications of VYJUVEK were associated with durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly with minimal reported adverse events. The GEM-3 trial was an intra-patient, double-blinded, multi-center, randomized, placebo-controlled study that met both its primary endpoint of complete wound healing at six months and its key secondary endpoint of complete wound healing at three months. VYJUVEK was well tolerated with no drug-related serious adverse events or discontinuations due to treatment-related events.
“Data from our GEM-1/2 trial and our GEM-3 trial, published in Nature Medicine and the
“Today’s landmark approval of VYJUVEK as the first redosable gene therapy ushers in a whole new paradigm to treat genetic diseases and is an important milestone for patients affected by DEB as well as their families and caregivers," said
VYJUVEK is expected to be available in
With this approval, the FDA issued the Company a Rare Pediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. The PRV program is designed to encourage the development of new drugs for the prevention or treatment of rare diseases.
“With the FDA approval of VYJUVEK, the DEB population has reached a monumental milestone in the treatment of this horrible disorder. Our hopes have now been realized for a safe and effective treatment for one of the most devastating symptoms of the disorder,” said
Outside of the US, the
Visit VYJUVEK.com for more information, including full Prescribing Information.
Investor Conference Call and Webcast
The Company will host an investor conference call and webcast to discuss the FDA’s approval of VYJUVEK on
To register and participate in the conference call, please go to: https://www.netroadshow.com/events/login?show=d32d1c5d&confId=51401. For those unable to listen to the live conference call, a replay will be available on the Investors section of the Company’s website at www.krystalbio.com.
VYJUVEK is a non-invasive, topical, redosable gene therapy 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 a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
IMPORTANT SAFETY INFORMATION
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
Warnings and Precautions
VYJUVEK gel must be applied by a healthcare provider.
After treatment, patients and caregivers should be careful not to touch treated wounds and dressings for 24 hours.
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 US Prescribing Information.
About Krystal Biotech, Inc.
Any statements in this press release about future expectations, plans and prospects for the Company including statements regarding the timing of the commercial availability of VYJUVEK in the
Investors and Media:
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/81a6404d-d9fb-4a1c-9d03-f8fb958c4cd8
Source: Krystal Biotech, Inc.