The Food and Drug Administration (FDA) has granted Fast Track designation to VX-880 (Vertex Pharmaceuticals) for the treatment of patients who have type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness.

VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy. The Company believes that VX-880 may be able to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

A single-arm, open-label phase 1/2 trial (ClinicalTrials.gov: NCT04786262) has been initiated to evaluate the safety, tolerability and efficacy of VX-880 infusion in adults who have T1D with severe hypoglycemia and impaired hypoglycemic awareness. The trial is expected to include approximately 17 adults with a clinical history of T1D (>5 years duration) who have had at least 2 episodes of documented severe hypoglycemia in the 12 months prior to enrollment.

Camillo Ricordi, MD, Professor of Surgery, Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami Miller School of Medicine, and Steering Committee Chair for the VX-880 clinical trial, commented: “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”


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The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

Reference

Vertex announces FDA Fast Track designation and initiation of a phase 1/2 clinical trial for VX-880, a novel investigational cell therapy for the treatment of type 1 diabetes. [press release]. Boston, MA: Vertex Pharmaceuticals; March 10, 2021.