Alfonso Sabater, MD, PhD, on Treating Ocular DEB With B-VEC


The associate professor of clinical ophthalmology at University of Miami discussed his experience using the gene therapy in a compassionate use program.

"Krystal Biotech reformulated the medication so that we could apply it into the eye. We designed some experiments to test the safety and efficacy in the eye in an animal model and then approached the FDA to try to get a compassionate use approval for our patient. After a few months, we got the approval and performed the surgery, this time on the right eye of our patient. And then we started applying the medication as an eye drop. It took a few months to recover after the surgery. In contrast to what we saw in the past, this time, we were not seeing blisters, we were not seeing new scarring coming back. And in fact, the eye recovered very well. After 6, 7 months, his vision was pretty good. And after 8 months, he had almost perfect vision.”

Krystal Biotech’s topical and redosable gene therapy beremagene geperpavec (B-VEC) was approved for for the treatment of dystrophic epidermolysis bullosa (DEB) in patients 6 months or older in May 2023 under the name Vyjuvek. While only approved for the treatment of B-VEC, the therapy has been used under a compassionate use approval in treating a single patient for ocular complications of DEB.

CGTLive spoke with Alfonso Sabater, MD, PhD, associate professor, clinical ophthalmology, and medical director, ocular surface program, and director, Corneal Innovation Lab, University of Miami, to learn more about his experiences in treating the patient with ocular complications of DEB in the compassionate use program.

Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech. May 19, 2023. Accessed May 19, 2023.
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