FDA grants fast track designation to stem cell therapy for type 1 diabetes
The FDA granted fast track designation to VX-880, a human stem cell-derived therapy for patients with type 1 diabetes, according to the agent’s manufacturer.
VX-880 (Vertex Pharmaceuticals) is a first-in-class, allogeneic, fully differentiated islet cell therapy designed to regulate glucose levels by restoring a patient’s pancreatic islet cell function, including insulin production.
A phase 1/phase 2 clinical trial for VX-880 has begun enrolling patients with type 1 diabetes with severe hypoglycemia and impaired hypoglycemic awareness.
“It’s a remarkable time for [type 1 diabetes] research efforts worldwide, as this investigational treatment enters the clinic,” Camillo Ricordi, MD, professor of surgery, director of the Diabetes Research Institute and Cell Transplant Center at University of Miami Miller School of Medicine, as well as steering committee chair for the VX-880 clinical trial, said in a press release. “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 [type 1 diabetes].”
The single-arm, open-label, multipart, sequential trial will assess the safety and efficacy of VX-880 at different doses. The trial will enroll about 17 participants, who will receive an infusion of VX-880 in addition to long-term immunosuppressive therapy to prevent immune rejection of pancreatic islet cells.
Preliminary trial sites enrolling patients include University of Miami Health System, University of Pennsylvania, and Massachusetts General Hospital, with more to be added by the end of the year, according to the manufacturer.
“Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets,” Bastiano Sanna, PhD, executive vice president and chief of cell and genetic therapies at Vertex, said in the release. “We continue to work with urgency to bring this innovative therapy to patients.”
FDA fast track designation helps to expedite development, review and potential approval of treatments for serious or life-threatening diseases.