FDA grants Orphan Drug Designation to TxCell's Col-Treg for treatment of autoimmune uveitis

Sep 15 2015

A first-in-man clinical study is planned to start in 2016, with top line results expected end 2017

TxCell SA (FR0010127662 – TXCL), a biotechnology company developing innovative, personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announces today that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted Orphan Drug Designation (ODD) 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. This followed the ODD 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 is currently approved for the patients that become refractory to corticosteroid compounds.

“The Orphan Drug Designation in both the US and EU 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 US and EU, 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.