Marta Figueiredo, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has approved Vyjuvek (beremagene geperpavec), Krystal Biotech’s gene therapy gel, for treating skin wounds in patients 6 months and older with dystrophic epidermolysis bullosa (DEB). Vyjuvek becomes the first re-dosable gene therapy approved by the FDA, and the only medicine approved…

Krystal Biotech’s gene therapy gel Vyjuvek (beremagene geperpavec) for dystrophic epidermolysis bullosa (DEB) has been granted priority review by the U.S. Food and Drug Administration (FDA). Priority review status is expected to shorten the review process for applications to six months from the standard 10 months. A decision…

The European Commission has approved Amryt Pharma’s Filsuvez (Oleogel-S10) for the treatment of skin wounds in adults and children, ages 6 months and older, with dystrophic epidermolysis bullosa (DEB) or junctional epidermolysis bullosa (JEB). The decision makes Filsuvez, a topical gel, the first-ever therapy approved for EB wounds.

Vyjuvek, a gene therapy gel (previously called B-VEC) by Krystal Biotech, safely and effectively improved wound healing in children and adults with recessive dystrophic epidermolysis bullosa (RDEB), according to data from a Phase 1/2 clinical trial. Importantly, resolved wounds remained closed for at least three months and up to eight…

The U.S. Food and Drug Administration (FDA) has rejected an application seeking approval of Filsuvez (Oleogel-S10) for treating skin wounds in people with junctional (JEB) and dystrophic epidermolysis bullosa (DEB). In its complete response letter to Amryt Pharma, the therapy’s developer, the FDA stated that the…

Transplanting a patient’s own genetically corrected skin grafts safely and effectively treated skin loss and ulcers affecting 80% of the body in a boy with a life-threatening form of generalized junctional epidermolysis bullosa (JEB), a study shows. Notably, these benefits were sustained for at least five years, highlighting that this…

The European Medicines Agency (EMA) is seeking the opinion of independent epidermolysis bullosa (EB) experts and patients before issuing its recommendation on the potential approval of Amryt Pharma’s topical gel Filsuvez (Oleogel-S10) to treat skin wounds in people with junctional EB (JEB) and dystrophic EB (DEB). Given…

With the new school year launching, the Dystrophic Epidermolysis Bullosa Research Association of America — better known as debra of America — is aiming to facilitate conversation and collaboration between families of children with epidermolysis bullosa (EB) and their teachers and classmates. To that end, debra is providing…

Dominant dystrophic epidermolysis bullosa (DEB) may lead to congenital deformities such as disproportionately short great (big) toes, according to a case report. In this rare case, the newborn also lacked skin in the lower part of both legs (a more common symptom of DEB), and with appropriate treatment his wounds…

Eloxx Pharmaceuticals announced ZKN-013 as its lead oral treatment candidate for recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB) caused by nonsense mutations in the COL7A1 gene. Preclinical safety and toxicology studies, required to secure U.S. Food and Drug Administration (FDA) approval for clinical trials of ZKN-013, are…

Excessive thickening of the skin on the soles of the feet (plantar keratoderma) is a common and serious complication of all subtypes of epidermolysis bullosa simplex (EBS), a study from Germany reports. This complication, appearing in early childhood, was significantly associated with pain, obesity, and limited mobility, negatively affecting employment…

Using exon skipping therapies may be an easy, affordable, and effective approach for people with epidermolysis bullosa (EB) with amenable mutations, an early study suggests. Such therapies would promote the production of a slightly shorter, but working version of the skin-related protein that is missing in people with EB, researchers said.