TxCell, a France-based biotechnology company developing innovative, personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announced today that the FDA’s Office of Orphan Products Development has granted Orphan Drug designation to TxCell’s Col-Treg, a personalized T cell immunotherapy using collagen-II specific regulatory T-cells, for the treatment of chronic non-infectious uveitis. The move followed the Orphan Drug designation already received for the product in Europe.
Autoimmune uveitis is a severe inflammatory condition of the eye, often resulting in permanent vision damage. Uveitis is one of the leading causes of blindness in the developed world. No treatment currently is approved for the patients that become refractory to corticosteroid compounds.
“The Orphan Drug designation in both the U.S. and E.U. is the latest significant step for the development of Col-Treg, a novel and promising therapeutic approach for autoimmune uveitis, resulting from TxCell’s ASTrIA platform. TxCell applied for and received Orphan Drug designation in the U.S. and E.U., which will allow us to expedite Col-Treg through development. This ultimately will benefit those suffering from autoimmune uveitis, a truly debilitating condition of the eye. TxCell plans to move Col-Treg into a first clinical study in 2016, with top line results expected end 2017,” said Stephane Boissel, CEO, TxCell.
The FDA Orphan Drug designation provides a special status to drugs and biologics intended to treat, diagnose or prevent rare diseases and disorders. Rare diseases and disorders are defined as affecting fewer than 200,000 people in the U.S. In particular, the designation provides for a seven-year marketing exclusivity period against competition as well as certain incentives, including federal grants and tax credits. It adds to the benefits of the E.U. Orphan Drug designation, which includes 10 years of market exclusivity from product launch as well as protocol assistance and possible exemptions or reductions in regulatory fees during development.