The U.S. Food and Drug Administration has granted accelerated approval to Tzield (teplizumab) for a new indication, delaying the decline of insulin production in pediatric patients ages 8 through 17 who have been recently diagnosed with Stage 3 type 1 diabetes. This is the first FDA-approved treatment for this indication.

“The FDA recognizes the large unmet need in patients with type 1 diabetes,” said Dr. Mahtab Niyyati, Acting Associate Director for Therapeutic Review of the Division of Diabetes, Lipid Disorders and Obesity. “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.”

Tzield was previously approved to delay the onset of Stage 3 type 1 diabetes in adults and pediatric patients 1 year of age and older with Stage 2 type 1 diabetes. The newly approved indication expands its use to children who have already progressed to Stage 3 disease, the stage at which blood glucose levels become clinically abnormal and insulin therapy is typically required.

The FDA granted the application under its accelerated approval pathway, based on evidence from a clinical trial showing that Tzield demonstrated a statistically significant effect on C-peptide levels, a surrogate endpoint reasonably likely to predict clinical benefit. A required postapproval study is ongoing to verify the clinical benefit.

The approved labeling includes a boxed warning regarding serious and life-threatening cases of viral reactivation, including Epstein-Barr virus (EBV) and cytomegalovirus (CMV) reactivation. The most common side effects reported with Tzield are vomiting, rash, increased liver transaminase, and headache. The drug is also associated with a reduction in white blood cells (leukopenia), including lymphopenia and neutropenia, which can increase the risk for certain infections.

Source: FDA Press Release