FDA approves Vyjuvek for dystrophic epidermolysis bullosa
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Key takeaways:
- The FDA approved Vyjuvek (beremagene geperpavec-svdt) for patients aged six months or older.
- The topical, redosable gene therapy for dystrophic epidermolysis bullosa produces copies of the COL7A1 gene.
The FDA has approved Vyjuvek (beremagene geperpavec-svdt), an investigational non-invasive, topical, redosable gene therapy for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
The redosable gene therapy is approved for patients aged six months or older with recessive or dominant dystrophic epidermolysis bullosa (DEB) and must be administered by a health care professional. The therapy, which is the only therapy approved by the FDA for the treatment of DEB, works by aiding the replication of the COL7A1 gene, which is responsible for producing the COL7 proteins that facilitate wound healing.
DEB, which is caused by mutations in the COL7A1 gene, is a disease that affects the skin and mucosal tissues. When applied to DEB wounds, Vyjuvek is designed to help patients’ skin create normal COL7 protein rather than mutations.
“This is a devastating disease,” M. Peter Marinkovich, MD, primary investigator of the GEM-3 trial, director of the Blistering Disease Clinic at Stanford Health Care and associate professor of dermatology at the Stanford University School of Medicine, said in a company press release. “Until now, doctors and nurses had no way to stop blisters and wounds from developing on dystrophic EB patient skin and all we could do was to give them bandages and helplessly watch as new blisters formed. VYJUVEK topical gene therapy changes all of this.”
The approval is based on data from the GEM-1/2 and GEM-3 clinical trials.
“Data from our GEM-1/2 trial and our GEM-3 trial, published in Nature Medicine and the New England Journal of Medicine, respectively, demonstrated the strength of both studies showing that Vyjuvek safely and effectively improved wound healing,” Suma Krishnan, president of research and development at Krystal Biotech, Inc. said in the release. “For so many years, all we have been able to offer DEB patients was palliative care, but now, based on the strength of the Company’s clinical trial data, there is a safe and effective FDA approved treatment.”
Perspective
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The approval of Krystal’s therapy B-VEC represents a number of “firsts” in gene therapy and demonstrates the expanding toolkit of gene therapy approaches to address serious diseases with high unmet medical need.
This would be the first gene therapy approved for a skin condition, the first approval of a gene therapy for rare genetic disease that uses a viral vector (Herpes Simplex Virus Type 1 — HSV1) other than [adeno-associated virus] or lentivirus, and the first approval of a topical, non-invasive gene therapy whose intent is to be redosable.
It represents the sixth gene therapy for a rare genetic disease approved by FDA and could be one of up to five gene therapies approved by the FDA for rare genetic diseases in 2023.
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