Krystal clear: FDA approves redosable gene therapy gel Vyjuvek

Krystal Biotech has won approval for Vyjuvek (beremagene geperpavec), a topical gel gene therapy to treat the skin condition dystrophic epidermolysis bullosa (DEB).

The handful of gene therapies on the market are single-dose injections or infusions, but the US Food and Drug Administration (FDA) approved the first ‘redosable’ gene therapy Friday, in the form of Krystal’s Vyjuvek.

Vyjuvek is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, which when administered topically to DEB wounds delivers two copies of the COL7A1 gene – the gene responsible for producing 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).

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Irina_drozd

“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 Krish Krishnan, CEO of Krystal Biotech.

“For Krystal, this is a transformative achievement that highlights our commitment to developing and commercializing novel therapies for patients with rare diseases and demonstrates Krystal’s capability as a fully-integrated company ready to launch and bring Vyjuvek to patients as quickly as possible and deliver additional transformative medicines to patients as we advance our pipeline.”

According to the FDA labeling information, drug substance and drug product through formulation and filling will occur at Krystal’s ‘Ancoris’ 4,500 square-foot facility in Pittsburgh, Pennsylvania. The firm also began construction of a second facility – named ‘Astra’ in 2020 – in nearby Findlay Township in 2020.

Vyjuvek is expected to be available in the United States in the third quarter of 2023.