News

AC-203 clinical trial enrollment starts for EBS patients

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 approximately 80 people ages 6 months and older with localized, severe, or intermediate EBS. To be eligible for participation, patients must…

Abeona planning potential US launch of pz-cel to treat RDEB

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…

CHMP recommends Vyjuvek OK for DEB patients from birth

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 gets $75M for DEB gene therapy Phase 3 trial

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…

Arthritis ointment diacerein heals chronic wounds in girl with RDEB

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…

Altered immunity discovered in study of adults with RDEB

Adults with recessive dystrophic epidermolysis bullosa (RDEB) have abnormal immune cell profiles in blood and skin samples, a study reveals. The patients also had high blood levels of pro-inflammatory fat-like lipids and immune signaling proteins. These findings “underscore the concept of RDEB as [a] genetic disorder with distressed immunometabolism/inflammation,”…

EB patients report high psychosocial health burden in interviews

In interviews, people with epidermolysis bullosa (EB) reported that living with these rare skin diseases has broad impacts on their psychosocial health. This burden was seen across physical, emotional, social, and functional domains, per the study, conducted in Europe. According to the researchers, the findings emphasize the importance of…