Sernova Biotherapeutics Receives FDA Orphan Drug Designation for Autologous Islet Transplantation for Prevention of Diabetes Due to Total Pancreatectomy

Executive Summary

• Sernova Biotherapeutics received FDA Orphan Drug Designation for autologous islet transplantation (AIT) using its Cell Pouch Bio-hybrid Organ to prevent Type 3c diabetes following total pancreatectomy.
• The designation provides potential regulatory benefits including seven years of U.S. market exclusivity upon approval, tax credits for qualified clinical testing, and waivers of certain FDA user fees.
• The company is preparing to initiate a clinical trial for this indication and plans to advance both its Type 1 and Type 3c diabetes programs in the coming months.

Key Details

• FDA granted Orphan Drug Designation specifically for autologous islet transplantation (AIT) to prevent diabetes resulting from total pancreatectomy.
• Sernova’s Cell Pouch Bio-hybrid Organ is designed to support the engraftment and long-term function of transplanted therapeutic cells, aiming to preserve patient insulin production without requiring immune suppression.
• Clinical trial preparation involves isolating the patient’s own insulin-producing islet cells from the removed pancreas, placing them in the Cell Pouch, and transplanting them back into the patient.
• Orphan Drug Designation criteria apply to conditions affecting fewer than 200,000 people in the U.S., granting potential market exclusivity and development incentives subject to successful review of detailed applications.
• Type 3c diabetes (pancreatogenic diabetes) is caused by pancreatic damage or surgical removal, impairing insulin production and requiring management similar to Type 1 diabetes.

Notable Quotes

• “Patients who require total pancreatectomy face a life-changing outcome: the immediate loss of pancreatic endocrine function and the risk of complex, insulin-dependent diabetes,” said Melena Bellin, MD, Co-Director of the Total Pancreatectomy and Islet Autotransplant Program at the University of Minnesota and a member of Sernova’s Clinical Advisory Board. “Autologous islet transplantation in Sernova’s Cell Pouch has the potential to preserve a patient’s own insulin-producing cells following pancreatic surgery, without the use of immune suppressing therapies.”
• “Receiving Orphan Drug Designation from the FDA is an important milestone for Sernova,” said Jonathan Rigby, Chief Executive Officer of Sernova. “Our primary focus remains advancing a functional cure for type 1 diabetes. At the same time, this designation provides Sernova a potential exclusive lead position in preventing type 3c diabetes. T3cD is a natural extension of our primary focus on T1D and we are planning to advance both of these programs in the coming months.”