FDA Awards Castle Creek $1.8 Million Grant to Support Pivotal Study in RDEB

Rare Daily Staff

The U.S. Food and Drug Administration Office of Orphan Products awarded Castle Creek Biosciences a $1.8 million research grant over four years to support the phase 3 clinical development of FCX-007, an experimental gene therapy for the treatment of recessive dystrophic epidermolysis bullosa.

Recessive dystrophic epidermolysis bullosa (RDEB) is a genetic and progressive condition caused by the deficiency of collagen type VII (COL7). Severe cases of this condition involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical problems. There is no cure or treatment approved in the United States.

FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing the cells, and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution.

The Congressionally funded Orphan Products Development Grants Program is designed to enhance the development of medical products for patients with rare diseases. RDEB is a progressive, devastatingly painful and debilitating, rare genetic disorder, and one of the most chronic and severe forms of DEB. RDEB causes painful blistering, wounds and scarring in response to trauma to the skin, including friction due to typical activities such as rubbing or scratching.

The FDA granted Orphan Drug designation for FCX-007 for the treatment of DEB. In addition, the agency granted FCX-007 Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations for the treatment of RDEB.