FDA Approves First Cellular Therapy for Type 1 Diabetes

The FDA has approved CellTrans’ Lantidra (donislecel), the first allogeneic (donor) pancreatic islet cellular therapy to treat patients with type 1 diabetes. It is approved for patients who unable to achieve target glycated hemoglobin.

Type 1 diabetes is a chronic autoimmune disease that requires daily administration of insulin. In 2021, there were about 8.4 million people worldwide with type 1 diabetes, and 1.4 million in the United States, according to an analysis from a study published last year in The Lancet Diabetes & Endocrinology. Many of these patients are undiagnosed, with 63% to 70% of deaths in those younger than 25 years estimated to be due to non-diagnosis. The investigators also projected that by 2040, there will be 13.5 million to 17.4 million people worldwide with type 1 diabetes.

The primary mechanism of action of Lantidra is believed to be the secretion of insulin by the infused allogeneic islet beta cells. It is administered as a single infusion into the liver portal vein. An additional infusion of Lantidra may be performed depending on the patient’s response to the initial dose.

Peter Marks, M.D., Ph.D.

“Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures,” Peter Marks, M.D., Ph.D., 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.”

The safety and effectiveness of Lantidra was evaluated in two single-arm studies with 30 patients with type 1 diabetes. Overall, 21 participants did not need to take insulin for a year or more, with 11 participants not needing insulin for one to five years and 10 participants not needing insulin for more than five years. Five participants did not achieve any days of insulin independence.

The most common adverse reactions included nausea, fatigue, anemia, diarrhea and abdominal pain. A majority of participants experienced at least one serious adverse reaction related to the procedure for infusing Lantidra into the hepatic portal vein and the use of immunosuppressive medications needed to maintain the islet cell viability