FDA Approves First Pancreatic Islet Cell Therapy for Adults with Type 1 Diabetes
The FDA has approved the first pancreatic islet cellular therapy for the treatment of type 1 diabetes. The treatment is made from deceased donor pancreatic cells and is intended to treat adults with type 1 diabetes who, despite extensive diabetes management, cannot reach average blood glucose levels because of repeated episodes of severe low blood sugar.
“Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research said in a press release. “Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels.”
Treatment is administered as a single infusion into the hepatic portal vein with an additional infusion being performed depending on the patient’s response to the first dose. According to the FDA’s press release, the “primary mechanism of action of [the therapy] is believed to be the secretion of insulin by the infused allogeneic islet beta cells.” This allows the infused cells to produce enough insulin so some patients with type 1 diabetes may no longer need insulin to control their blood sugar levels.
The approval follows two non-randomized, single-arm studies that tested the efficacy and safety of the treatment. In total, 30 participants with type 1 diabetes and hypoglycemic unawareness (the inability to detect if blood glucose is dropping in a patient) received at least one infusion of treatment and a maximum of three infusions.
Of the total, 21 participants did not need to take insulin for a year or more. Further, 11 participants did not need insulin for 1 to 5 years, and 10 participants for more than 5 years. The remaining five participants did not achieve any number of days of insulin independence.
Adverse reactions varied with each patient and depended on the number of infusions they received and the length of time they were followed. The most common reported were nausea, fatigue, anemia, diarrhea, and abdominal pain. Some serious adverse reactions required the patient to discontinue use of immunosuppressive medications, which results in the loss of islet cell function and insulin independence.
FDA approves first cellular therapy to treat patients with type 1 diabetes. News release. US Food and Drug Administration; June 28, 2023. Accessed July 18, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes