Tzield (teplizumab) accepted for priority review in the US for young children with stage 2 type 1 diabetes – Sanofi
The FDA has accepted for priority review the supplemental biologic license application (sBLA) for Tzield (teplizumab-mzwv) to expand the current age indication from eight years and above, to as young as one year old and above to delay the onset of stage 3 type 1 diabetes (T1D) in patients diagnosed with stage 2 T1D. The sBLA is supported by the positive interim one-year data from the ongoing PETITE-T1D phase IV study (clinical study identifier: NCT05757713), evaluating the safety and pharmacokinetics of Tzield in young children. The PDUFA target action date for the FDA decision is 29 April 2026.
“This priority review emphasizes the urgent need for innovative therapies like Tzield which has the potential to prevent the natural progression of T1D by delaying the loss of endogenous insulin production. This might be particularly significant in this young population, as it is well documented that the autoimmune attack that drives this disease in many cases, begins, early in life,” said Christopher Corsico, Global Head of Development at Sanofi. “If approved, Tzield could represent an important advance for delaying the onset of stage 3 type 1 diabetes in early childhood, which would benefit patients and caregivers alike.”
Interim data for the PETITE-T1D phase IV study was presented at the 51st Annual Conference of the International Society for Pediatric and Adolescent Diabetes and simultaneously published in Diabetologia.
Priority review is given to regulatory applications seeking approval for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
The safety and efficacy of Tzield in the PETITE-T1D population has not been approved by any regulatory authority.
About PETITE-T1D
PETITE-T1D (clinical study identifier: NCT05757713) is an ongoing phase IV single-arm, non-randomized, open-label, multicentre study designed to assess the safety and pharmacokinetics of Tzield in children under eight years diagnosed with stage 2 T1D. Stage 2 T1D is defined by the presence of two or more T1D-related autoantibodies and dysglycaemia. The study has enrolled 23 participants. The regimen consists of an intravenous infusion of Tzield once daily for 14 consecutive days. The study duration for each individual may last up to 26 months for follow up and monitoring.
Citation: Safety and pharmacokinetics of teplizumab in children less than 8 years of age with stage 2 type 1 diabetes. Authors: Stephen E Gitelman, Kimber Simmons, Jennifer L Sherr. Diabetalogia 2025 Nov 6. doi: 10.1007/s00125-025-06586-1
