FDA
Tzield approved to delay onset of stage 3 type 1 diabetes in children as young as one year of age
On April 20, 2026, FDA approved an expanded indication for Tzield (teplizumab‑mzwv) for children aged one year and older with stage 2 type 1 diabetes (T1D), aiming to delay progression to stage 3 disease, when diabetes becomes clinically apparent. Previously approved for patients eight years and older, Tzield is the first disease‑modifying therapy shown to alter the natural history of autoimmune T1D.
The FDA decision was supported by one‑year data from the phase 4 PETITE‑T1D study, a single‑arm, open‑label, multicenter trial evaluating safety and pharmacokinetics in very young children with stage 2 T1D. The study enrolled 23 participants under eight years of age, all with two or more T1D‑related autoantibodies and dysglycemia. Participants received intravenous Tzield once daily for 14 consecutive days and were followed for up to 26 months, including screening and post‑treatment monitoring.
In an interim analysis, 2 treated patients progressed to stage 3 type 1 diabetes, with an estimated 89.6% probability of remaining progression‑free (95% confidence interval, 64.3 to 97.3).
Pharmacokinetic analyses showed that when infusion time was extended from 30 minutes to 2 hours, maximum teplizumab concentrations in children aged 1 to under 8 years were comparable to those observed in patients aged 8 years and older.
Among pediatric patients younger than 8 years old, the most frequently reported adverse events were vomiting and diarrhea.
By enabling immune intervention before symptomatic hyperglycemia develops, the expanded approval positions clinicians to delay insulin dependence during a particularly vulnerable period of childhood.
“This is especially important because these children are often at the highest risk of progressing quickly and without warning," said Kimber Simmons, MD, MS, Associate Professor of Pediatrics at Barbara Davis Center in Colorado. "Delaying the onset of stage 3 type 1 diabetes during the years when management is often most difficult because of a child’s small size and dependence on caregivers could have a truly meaningful impact for families."
Sources:
Sanofi. (2026, April 22). Press release. Sanofi's Tzield approved in the US to delay the onset of stage 3 type 1 diabetes in young children
Tzield (teplizumab-mzwv). [Package insert]. https://products.sanofi.us/tzield/tzield.pdf Revised April 2026.