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The European Commission has approved the topical gene therapy Vyjuvek (beremagene geperpavec) to treat wounds, starting at birth in people with dystrophic epidermolysis bullosa (DEB) who have disease-causing mutations in the COL7A1 gene. The decision, which applies to all European Union countries, and includes Iceland, Norway, and Liechtenstein,…

The U.S. Food and Drug Administration (FDA) has approved the cell-based gene therapy prademagene zamikeracel, previously pz-cel or EB-101, for treating wounds in adults and children with recessive dystrophic epidermolysis bullosa (RDEB). The therapy’s developer Abeona Therapeutics will market it under the brand name Zevaskyn. According to Abeona,…

People with severe recessive dystrophic epidermolysis bullosa (RDEB) are more likely to develop disease-related complications, and at a younger age, than those with milder or more localized disease types, a U.K. study suggests. Complications include skin cancer, swallowing and feeding difficulties, with patients commonly needing procedures to improve feeding…

Long-term treatment with Filsuvez (birch triterpenes) was well tolerated and associated with sustained reductions in wound burden for people with dystrophic epidermolysis bullosa (DEB) and junctional epidermolysis bullosa (JEB). That’s according to final two-year data from the open-label phase (OLP) of the Phase 3 EASE trial…

Note: This story was updated April 3, 2025, to correct that enrollment criteria in Europe differs from criteria at other sites. A Phase 2/3 clinical trial testing AC-203 (diacerein 1% ointment) in people with epidermolysis bullosa simplex (EBS) is recruiting participants. The trial, EBShield (NCT06073132), seeks to enroll…

Abeona Therapeutics is preparing for the potential launch of pz-cel (prademagene zamikeracel) to treat recessive dystrophic epidermolysis bullosa (RDEB), after the U.S. Food and Drug Administration (FDA) initiated discussions on the medication label and post-marketing requirements, according to a company press release. Last fall, the…

People with epidermolysis bullosa (EB) experience a deterioration in oral health, which is linked to poor health-related quality of life. That’s according to a study in Germany, which also found patients with dystrophic EB had poorer oral health and worse oral health-related quality of life when compared with…

A committee of the European Medicines Agency is recommending approving Vyjuvek (beremagene geperpavec), a gene therapy from Krystal Biotech, to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). The opinion by the Committee for Medicinal Products for Human Use (CHMP) will now go to…

Castle Creek Biosciences has secured $75 million in financing to support a Phase 3 trial testing its gene therapy to promote wound healing in people with dystrophic epidermolysis bullosa (DEB). The royalty financing agreement for Castle Creek‘s treatment, D-Fi, was led by Ligand Pharmaceuticals, which made…

France-based pharma company Theravia has obtained rights to an experimental molecule that may be used to treat epidermolysis bullosa (EB). The molecule, known as DCN-tCRK, was discovered by scientists at Tampere University, in Finland. Under the new licensing agreement, Theravia will be in charge of further drug development…

The stem cell therapy CORDStrom, in the pipeline at Inmune Bio, was found in a clinical trial to reduce itch and pain in children — from infants to teenagers — with intermediate and severe recessive dystrophic epidermolysis bullosa (RDEB), new data show. The treatment candidate also reduced disease…

A topical ointment containing diacerein, an arthritis medication, promoted wound healing in a 5-year-old girl with hard-to-treat recessive dystrophic epidermolysis bullosa (DEB), a case study reports. “These data characterize diacerein as a potential candidate for improving wound healing in RDEB through its impact on inflammatory [cells],” researchers wrote. The…