Breakthrough Clinical Results
Krystal Biotech announced that Japan's Ministry of Health, Labour and Welfare (MHLW) approved VYJUVEK® (beremagene geperpavec-svdt) for treating dystrophic epidermolysis bullosa (DEB) wounds in patients from birth. VYJUVEK is the first genetic medicine approved in Japan for DEB and allows for home or healthcare setting administration. The approval is based on a comprehensive clinical dataset, including results from a Japanese open-label extension study showing successful wound closure. The company expects to launch VYJUVEK in Japan by the end of 2025.
Key Highlights
- VYJUVEK® (beremagene geperpavec-svdt) received marketing authorization from Japan's MHLW for treating DEB wounds from birth.
- VYJUVEK® is the first genetic medicine approved in Japan for DEB and offers home administration.
- The Japanese approval was based on a comprehensive clinical dataset mirroring results from the US Phase 3 study.
- Krystal Biotech expects to launch VYJUVEK in Japan by the end of 2025.
Incidence and Prevalence
Latest Estimates of Incidence and Prevalence of Dystrophic Epidermolysis Bullosa
Dystrophic epidermolysis bullosa (DEB) is a rare genetic skin disorder caused by COL7A1 sequence variations that result in type VII collagen deficits. Based on available research, comprehensive global incidence and prevalence data for DEB are not specifically documented in recent literature.
Available Epidemiological Data
A 2019 study examining 644 enrolled patients from 17 sites in the United States and Canada found that: - 283 patients (43.9%) had Recessive dystrophic epidermolysis bullosa (RDEB) - 104 patients (16.2%) had Dominant dystrophic epidermolysis bullosa (DDEB)
Earlier research from 2014 indicated that dominant forms account for approximately 40% of dystrophic epidermolysis bullosa cases.
Clinical Significance and Complications
While global epidemiological metrics are not specifically quantified, the clinical significance of this condition is well-documented. The 2023 publication highlights that cutaneous squamous cell carcinoma (CSCC) is a common complication of RDEB, observed in 90% of patients with the severe subtype of RDEB by 55 years of age.
Current Research Focus
The available literature focuses primarily on: - Clinical manifestations - Diagnostic approaches - Complications - Treatment challenges
Rather than population-based epidemiological metrics, current research emphasizes the clinical characteristics and management of this condition.
Summary
DEB is consistently described as a rare condition across multiple sources. The condition is categorized into two main subtypes - RDEB and DDEB - with recessive forms appearing more common in clinical populations based on the 2019 study data. However, specific global epidemiological data regarding incidence or prevalence rates for either DDEB or RDEB is not available in recent literature.
Drug used in other indications
VYJUVEK® (beremagene geperpavec-svdt) Clinical Trials Beyond DEB
Based on the available information, there is no evidence that VYJUVEK® (beremagene geperpavec-svdt) is currently being trialed for any indications other than Dystrophic epidermolysis bullosa (DEB).
VYJUVEK® is the first FDA-approved topical gene therapy specifically for DEB. It utilizes a genetically modified HSV-1 (herpes simplex virus 1) vector to express human COL7 in patients with DEB. The therapy received its first approval in the US in May 2023 for "the treatment of wounds in patients ≥ 6 months of age with dystrophic epidermolysis bullosa with mutation(s) in the COL7A1 gene."
The medication is described as a topically applied, redosable, live, replication defective herpes simplex virus-1 (HSV-1) vector-based gene therapy developed by Krystal Biotech to deliver functional human collagen type VII alpha 1 chain (COL7A1) genes in patients with both dominant and recessive forms of dystrophic epidermolysis bullosa.
A Marketing Authorization Application for beremagene geperpavec in Europe was planned for the second half of 2023.
Clinical trials for DEB have demonstrated that VYJUVEK® increases the probability of complete wound healing compared to placebo.
Since there is no information about VYJUVEK® being tested for other indications beyond DEB, details about intervention models for such trials cannot be provided.
Krystal Biotech's FDA-Approved Indications (2021-2024)
Based on a comprehensive review of Krystal Biotech, Inc.'s regulatory approvals over the past three years (2021-2024), the company has received FDA approval for the following indications:
In May 2023, Krystal Biotech received FDA approval for VYJUVEK™ (beremagene geperpavec-svdt), which is indicated for the treatment of dystrophic epidermolysis bullosa (DEB). This represents a significant milestone as VYJUVEK™ is the first and only FDA-approved treatment for DEB, a rare and serious genetic condition affecting the skin.
VYJUVEK™ utilizes Krystal's proprietary HSV-1 based gene therapy platform to deliver functional copies of the COL7A1 gene directly to the skin. This topical gene therapy addresses the underlying genetic cause of DEB by enabling the production of functional collagen VII protein, which is essential for skin integrity.
Beyond this groundbreaking approval for DEB, the available information does not indicate that Krystal Biotech has received FDA approval for any other therapeutic indications during the specified timeframe (2021-2024). The company continues to focus on developing additional candidates using their innovative gene therapy platform for various genetic diseases, but these have not yet received regulatory approval within the timeframe specified.
The approval of VYJUVEK™ represents a major advancement in the treatment landscape for patients with DEB, who previously had no approved therapies targeting the root cause of their condition.