WASHINGTON, D.C. — The U.S. Food and Drug Administration has expanded the approved use of Tzield (teplizumab) to children ages 8 to 17 who have been recently diagnosed with Stage 3 type 1 diabetes, marking the first approved therapy aimed at delaying the decline of insulin production after diagnosis.
The accelerated approval, granted June 12, broadens the drug’s role in type 1 diabetes treatment and could provide pediatric patients with additional time before significant loss of pancreatic insulin-producing function.
Tzield was previously approved to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients with Stage 2 disease. The new indication allows the therapy to be used after progression to Stage 3 diabetes in certain recently diagnosed children and adolescents.
The FDA based its decision on results from a controlled clinical trial demonstrating a statistically significant effect on C-peptide levels, a biomarker used to measure insulin production. Agency officials concluded the endpoint was reasonably likely to predict clinical benefit.
“The FDA recognizes the large unmet need in patients with type 1 diabetes,” Dr. Mahtab Niyyati, acting associate director for therapeutic review in the FDA’s Division of Diabetes, Lipid Disorders and Obesity, stated. “Based on robust evidence of safety and effectiveness, this accelerated approval provides a chance for pediatric patients with recently diagnosed Stage 3 type 1 diabetes to alter the course of their disease.”
Because the approval was granted under the FDA’s accelerated approval pathway, the manufacturer is required to complete an ongoing post-approval study to confirm clinical benefit.
The agency noted that Tzield carries a boxed warning regarding serious and potentially life-threatening viral reactivation, including Epstein-Barr virus and cytomegalovirus infections.
Common adverse effects reported with the treatment include vomiting, rash, headache, and elevated liver enzyme levels. The therapy also can reduce white blood cell counts, including lymphocytes and neutrophils, potentially increasing susceptibility to certain infections.
The approval represents the first FDA-authorized treatment specifically indicated to delay the decline of insulin production in pediatric patients recently diagnosed with Stage 3 type 1 diabetes.
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