FDA clears IND application for stem cell therapy to treat type 1 diabetes
The FDA cleared an investigational new drug application for VX-880, a stem cell-derived therapy for patients with type 1 diabetes, according to the agent’s manufacturer.
VX-880 (Vertex Pharmaceuticals) is an allogeneic, human stem-cell derived, fully differentiated pancreatic islet cell therapy. The agent is designed to reestablish the patient’s ability to “regulate glucose levels by restoring pancreatic islet cell function, including insulin production,” according to the manufacturer.
The IND clearance will allow Vertex to begin a phase 1/phase 2 study of VX-880. The single-arm, open-label study will enroll about 17 patients with type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia.
The multipart, sequential clinical trial will assess the safety and efficacy of VX-880 at different doses. Trial participants will receive an infusion of VX-880 in addition to long-term immunosuppressive therapy to prevent immune rejection of pancreatic islet cells.
“As we celebrate the 100th anniversary of the discovery of insulin this year, we are excited to bring a first-in-class cell therapy to the clinic with the potential to meaningfully impact people living with [type 1 diabetes],” Bastiano Sanna, PhD, executive vice president and chief of cell and genetic therapies at Vertex, said in a company-issued press release. “We look forward to getting our clinical program underway and testing our unique approach of replacing pancreatic islet cells — which are destroyed in people with type 1 diabetes — with our stem cell-derived, fully differentiated insulin-producing pancreatic islet cells.”
Vertex plans to begin the trial in the first half of 2021.