Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264
Friday, March 10, 2023
Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes (T1D). The VX-264 programme does not require the use of immunosuppression, which may broaden the population of people with T1D that this investigational therapy could reach.
VX-264 is an investigational cell therapy in which allogeneic human stem cell-derived islets are encapsulated in a channel array device designed to shield the cells from the body’s immune system. VX-264 is designed to be surgically implanted and is currently being evaluated for patients with T1D.
The clinical trial is a Phase 1/2, single-arm, open-label study in patients who have T1D. This will be a sequential, multi-part clinical trial to evaluate the safety, tolerability and efficacy of VX-264 in patients with T1D.
