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First gene therapy for DEB patients approved in the US

Approval for gene therapy gel for EB in the US

On 19 May 2023, the US Food and Drug Administration (FDA) made a positive decision to approve the gene therapy gel VYJUVEK from Krystal Biotech.

Dystrophic EB patients usually live with a severe form of the disease that affects the skin and the mucous membranes and is caused by mutations in the collagen 7 gene. The drug with the name VYJUVEK (also known as B-VEC) is a topical gel that locally remedies the genetic cause of dystrophic EB by adding functional copies of the collagen 7 gene to patients' skin cells. It is a redosable gene therapy using an engineered herpes simplex virus type 1 (HSV-1) for the delivery of the gene copies.

VYJUVEK is the first drug and first in vivo gene therapy approved for EB patients in the US. The Filsuvez gel from Amryt, has already been approved in Europe, but not yet in the US.
The VYJUVEK applies to patients six months or older with recessive or dominant dystrophic EB in the US. The gel is applied to selected wounds once a week - one wound at a time until wound closure. The FDA has accepted the use of VYJUVEK by healthcare professionals in a clinic or at home.

The new gene therapy is expected to be available for patients in the US by the end of 2023. Outside the US, the European Medicines Agency has already granted VYJUVEK orphan drug designation and PRIME (PRIority MEdicines) approval for treating DEB patients. The company expects the official approval process to begin in the second half of 2023, with possible approval in 2024. The gel is not expected to be accessible in Europe until 2025 when market availability is targeted. After that, negotiations with the respective health insurance companies regarding reimbursement will begin.

Krystal Press release

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