Teplizumab Approval Expanded to Children >1 Year with Stage 2 Type 1 Diabetes (T1D)  

Prepared on behalf of PES Drugs and Therapeutics Committee by Pranav Gupta, MD and edited by Neha Patel, DO. 

Teplizumab is a humanized anti-CD3 monoclonal antibody that mitigates autoimmune beta-cell destruction by increasing regulatory T cells and inducing exhaustion of CD4+ and CD8+ T-cells, thereby reducing autoreactive T-cell activity and delaying disease progression.1   

Teplizumab was initially approved in the US in November 2022 to delay the onset of Stage 3 T1D in adults and children >8 years with Stage 2 T1D.2 In April 2026, the FDA expanded approval to include patients as young as 1 year old.3 

The phase 4 PETITE-T1D trial (NCT05757713), a 2-year, open-label, single-arm study in 23 children <8 years with stage 2 T1D, supported this approval. After a 14-day teplizumab course —administered as 2‑hour infusions in children aged 1–8 years due to peak drug concentrations (vs. 30 minutes in older patients)— most adverse events were mild or moderate, including vomiting, rash, leukopenia, and diarrhea;13% discontinued treatment due to adverse events Two participants had maculopapular rash and deep vein thrombosis at PICC line. Two participants progressed to stage 3 T1D, with estimated 89.6% remaining progression-free at ~ 1 year.4 

References: 

  1. Lledó-Delgado A, Preston-Hurlburt P, Currie S, et al. Teplizumab induces persistent changes in the antigen-specific repertoire in individuals at risk for type 1 diabetes Clin Invest. Aug 132024;134(18)doi:10.1172/jci177492 
  2. Keam SJ. Teplizumab: First Approval. Drugs. Apr 2023;83(5):439–445. doi:10.1007/s40265-023-01847-y
  3. Press Release: Sanofi’s Tzield approved in the US to delay the onset of stage 3 type 1 diabetes in young children. Updated 04/22/2026. Accessed 05/04/2026, https://www.sanofi.com/en/media-room/press-releases/2026/2026-04-22-05-05-00-3278650 
  4. Gitelman SE, Simmons K, Sherr JL, et al. Safety and pharmacokinetics of teplizumab in children less than 8 years of age with stage 2 type 1 diabetes. Diabetologia. Feb 2026;69(2):330–342. doi:10.1007/s00125-025-06586-1

 

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