Janet Stewart, MSc,  —

The first participant has been enrolled in a Phase 2 clinical trial testing AC-203 for the treatment of inherited epidermolysis bullosa (EB), the trial’s sponsor, TWi Biotechnology, announced. AC-203 (also known as CCP-020) is a proprietary topical formulation of 1% diacerein, a compound able to inhibit factors that contribute to…

U.S. residents with some types of inherited epidermolysis bullosa (EB) can now participate in Amryt Pharma‘s Phase 3 trial testing AP101 (Oleogel-S10), an experimental treatment for EB. This comes in the wake of the U.S. Food and Drug Administration granting investigational new drug (IND) clearance to AP101. The…

The U.S. Food and Drug Administration has cleared Krystal Biotech‘s investigational new drug application for KB103, a topical gene therapy for dystrophic epidermolysis bullosa (DEB). The company can now start enrolling patients for a clinical trial to test the experimental therapy. KB103 is based on the company’s…

A bone-anchored hearing aid was implanted in a patient with epidermolysis bullosa (EB) who had mixed hearing loss, according to a case report. There was minimal damage to the surrounding skin, which is generally seen with conventional hearing aids in EB patients. The case report, “Successful…

A Phase 2/3 clinical trial testing diacerein in the form of a cream applied to the skin showed that it could significantly reduce the number of blisters in patients with epidermolysis bullosa simplex (EBS). No treatment-related adverse events were reported. The results of the small Austrian trial were published…

Researchers found two mutations in a gene that carries the instructions for the protein plectin in a patient with epidermolysis bullosa simplex (EBS). Plectin helps maintain the integrity of the skin, and the research team believes the mutations they found might be the cause of the disease in this patient. The…

Orphan Drug Designation was recently granted to ProQR Therapeutics’ investigational therapy QR-313 for the treatment of dystrophic epidermolysis bullosa (DEB) by the European Medicines Agency (EMA). Orphan Drug Designation (ODD) in Europe is granted to drug candidates intended for the treatment, prevention or diagnosis of diseases that…

Low bone mineral density, a measure of bone health, was associated with low vitamin D levels and more severe disease in children with epidermolysis bullosa (EB), an Italian study found. The authors recommended more studies to investigate the possible benefits of vitamin D supplements to improve bone health in affected…

Cannabinoid therapy developer InMed and French tissue engineering company ATERA have teamed up to develop 3-D human skin models with tissue taken from epidermolysis bullosa (EB) patients. The models will be used to test InMed’s candidate therapy INM-750 in the lab before moving to clinical trials in…

The European Medicines Agency (EMA) recently granted Tarix Orphan’s lead investigational product TXA127 orphan drug status for the treatment of epidermolysis bullosa (EB). Orphan drug status is granted by regulatory agencies to drugs in development for the treatment of rare diseases, and where the pharmaceutical company will profit…