New Hope for Children With Devastating Skin Condition

New Hope for Children With Devastating Skin Condition

February 7, 2013

It begins with blisters—on the hands, the feet, the knees. In its  severest form the blisters are present over the entire body, in the mouth, in the digestive tract. Birth itself can be a trauma for those with this genetic condition, leaving infants with entire swaths of missing skin. Scarring usually results. Fused appendages. Permanently vanished nails.

The condition is called Dystrophic Epidermolysis Bullosa (DEB), and researchers estimate that it affects 6.5 per million newborns in the United States. Recently Shire entered this meaningful research space when it announced its intention to buy Lotus Tissue Repair, a biotech company now in the late pre-clinical stages of developing a protein replacement therapy for DEB. The goal, of course, is to create access to life-changing treatment for patients who at the moment, have none.

Dr. Mark de Souza, former CEO of Lotus Tissue Repair, will work closely with Shire over the coming year to ensure a smooth transition of the program. At the time of the announcement, Dr. de Souza called Shire “a great home for our rC7 program,” while John Dart, the COO of a patient organization noted that “it is immensely encouraging that the future of this exciting potential therapy is in safe and experienced hands. We have worked closely with Lotus and look for- ward to doing so with Shire as we work to get protein therapy into the clinic.”

These developments complement work already underway in Shire where we have recently initiated a Phase 3 study of a skin substitute therapy to treat non-healing wounds in people with Epidermolysis Bullosa (EB). The product is created by growing human skin cells on a mesh that can be absorbed by the body.

In all, these are promising developments for those with EB and DEB, and underscores Shire’s commitment to developing new treatments for those with rare and life-altering conditions who currently have few if any treatment options.