Shire plc (Nasdaq: SHPG), which has a facility in San Diego, on Friday announced the initiation of a Phase 3 study designed to evaluate the efficacy and safety of ABH001, its dermal substitute therapy, for the treatment of non-healing wounds in patients with epidermolysis bullosa (EB).
EB is a group of rare genetic skin disorders that begin to manifest at birth or early childhood and occur in approximately 19 per 1 million live births in the United States.
"People affected by EB suffer skin blisters and almost constant, acute pain and scarring," said the study's principal investigator, H. Alan Arbuckle, M.D., Section Head Pediatric Dermatology Kaiser Permanente Colorado, Wound Care Consultant, Epidermolysis Bullosa Center of Excellence, The Children's Hospital, Aurora Colo. "The current standard of care is daily wound care, bandaging and pain management. I am excited to be involved in testing the efficacy and safety of ABH001 as a potential treatment option for these patients."
ABH001 for EB has been granted an orphan drug designation in the United States and EU, and has also received "fast track" designation from the U.S. Food and Drug Administration (FDA). In addition, the European Medicines Agency's Pediatric Committee has agreed on a pediatric investigation plan for ABH001 for the treatment of EB.
The new Phase 3 study is a multi-site, prospective, randomized, open-label, intra-subject controlled trial evaluating the efficacy and safety of ABH001 to initiate healing and reduce the wound surface area of selected stalled, chronic cutaneous wounds associated with generalized EB.
Approximately 20 subjects with generalized EB aged three years and older are planned to enroll in the trial, which is targeted to be conducted in 10 to 15 sites across the United States, Europe and Canada. The study will comprise ABH001 applications sufficient to cover the surface area of the wound, applied topically every four weeks with protocol-specified dressings until healed or for up to 24 weeks.