FDA update allows at-home gene therapy for all DEB patients
Vyjuvek wound gel now approved for newborns, patient and caregiver use
The U.S. Food and Drug Administration (FDA) approved an update to the prescribing information of Vyjuvek (beremagene geperpavec), a gene therapy gel for treating wounds in people with dystrophic epidermolysis bullosa (DEB), that allows its use in all patients and permits self-administration at home.
The treatment was previously approved for DEB patients aged 6 months and older. The update expands the approval to cover babies with DEB from birth and to make administering it more convenient, according to the therapy’s developer, Krystal Biotech.
Vyjuvek is available as a biological suspension that’s mixed with a gel before being applied to the skin as drops onto the treated wound(s). Previously, the therapy could only be administered by a medical professional, but with the new update, patients and caregivers can administer it themselves at home. Vyjuvek should not be administered by anyone who is pregnant, and pregnant people should avoid contact with treated wounds.
The update also allows more flexibility with bandaging Vyjuvek-treated wounds. Previously, dressings of treated wounds had to be left for 24 hours, but now they can be removed along with the next scheduled bandage change.
“We believe these changes further reinforce VYJUVEK’s leadership position as the most flexible, convenient, and disease-modifying medicine to treat DEB patients in the United States,” Krish Krishnan, chairman and CEO of Krystal, said in a company press release.
Update hailed as ‘enormous, positive change’
DEB is caused by mutations in the gene COL7A1, which provides instructions to make an important structural protein that helps skin retain its integrity. A deficit of this protein leads to chronic wounds that don’t heal well. Vyjuvek is designed to deliver two healthy copies of the COL7A1 gene to skin cells, restoring production of the protein and promoting wound healing.
“By providing patients and their caregivers the ability to apply VYJUVEK themselves, we have made it easier for patients to integrate VYJUVEK into their daily routines and lifestyle,” Krishnan said. “Overall, we believe that these advancements solidify VYJUVEK as the standard of care for all DEB wounds, regardless of wound size and severity, and will further improve compliance.”
The company said the update is based on real-world data collected since Vyjuvek was commercially launched in the U.S. in 2023 and on recently published clinical trial data tracking long-term outcomes with the therapy.
“The updates to the VYJUVEK label are yet another significant and impactful step forward for all those living with DEB,” said Brett Kopelan, executive director of the EB advocacy group debra of America. “Enabling caretakers to apply VYJUVEK during their standard of care regimen is an enormous positive change allowing for increased convenience without sacrificing safety and “will only increase the quality of life of those living with this challenging disorder and that is exactly what our community needs.”
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