麻豆传媒

The FDA has approved the first allogeneic pancreatic cell treatment for type 1 diabetes, the .

Dubbed donislecel (Lantidra), the pancreatic islet cellular therapy is indicated for adults with "brittle" type 1 diabetes marked by an inability to achieve target HbA1c due to recurrent episodes of severe hypoglycemia even with intensive diabetes management. The novel treatment is thought to work through the secretion of insulin via the infused islet beta cells from deceased donors.

"Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, in a statement. "[This] 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."

Donislecel is administered by a single infusion into the hepatic portal vein. Based on patient response to the initial dose, an additional infusion may be warranted. And just like with an organ transplant, long-term immunosuppression is required to maintain the therapy's viability, according to developer CellTrans.

The long-awaited approval arrives more than 2 years after an FDA advisory committee voted 12-4 in favor of approval for this patient population.

In the two small single-arm studies underpinning the approval -- the phase I/II UIH-001 study and the phase III UIH-002 study -- 70% (21 of 30) of the participants were able to achieve at least a full year of insulin independence while maintaining glycemic control following islet transplantation. Insulin independence was defined as not using insulin while keeping HbA1c at 6.5% or lower 2 weeks after transplant. Study patients received up to three infusions.

Ten of the 21 patients achieved insulin independence for 5 years and 11 patients for 1 to 5 years. However, five patients never became insulin independent at any time during the average 6.5-year follow-up, one of whom received two transplants.

The treatment didn't come without risk too, with a total of 1,319 adverse events (75 severe) reported during the first year after transplantation, eight of which were life-threatening. One patient in the phase III trial died due to multiorgan failure from an infection 592 days after initial transplant, which the developer deemed "probably related to immunosuppression/study drug."

The most common adverse events during the studies included nausea, fatigue, anemia, diarrhea, and abdominal pain. Most patients also experienced at least one serious adverse reaction related to the procedure for infusing donislecel into the hepatic portal vein and the use of immunosuppressive medications, some serious enough to lead to discontinuation of the immunosuppressants, prompting loss of islet cell function and insulin independence.

The approval marks an era moving toward more functional "cures" for type 1 diabetes. Earlier this week at the American Diabetes Association Scientific Sessions, Vertex Pharmaceuticals released additional data on its VX-880 cell therapy, which works by an infusion of stem cell-derived beta cell therapy. Here, two patients with type 1 diabetes achieved full insulin independence.

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