FDA grants fast Track Designation to human stem cell therapy for Type 1 Diabetes

The U.S. Food and Drug Administration (FDA) has 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. The company has initiated a clinical trial for VX-880 in patients who have type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness.

It is a novel first-in-class, allogeneic, fully differentiated islet cell therapy designed to regulate blood sugar levels by restoring patient's pancreatic islet cell function, including insulin production.

"This program has its roots in the groundbreaking work that began in Dr. Doug Melton's lab, progressed at Semma Therapeutics, and has been accelerated and brought to the clinic by the team at Vertex," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. "Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets.

We are very pleased to have received FDA's Fast Track Designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients."

"It's a remarkable time for T1D research efforts worldwide, as this investigational treatment enters the clinic," said Camillo Ricordi, M.D., 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.

"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."

The first clinical trial sites at the University of Miami Health System, the University of Pennsylvania and Massachusetts General Hospital are open for enrollment, and additional sites will be activated this year. To learn more visit clinicaltrials.gov.