The US Food and Drug Administration has approved an expanded indication for teplizumab-mzwv (Tzield) to delay the onset of stage 3 type 1 diabetes (T1D) in pediatric patients aged 1 year and older with stage 2 T1D, according to a press release from Sanofi. The therapy had previously been approved for patients aged 8 years and older.

The approval was supported by 1-year data from the PETITE-T1D Phase 4 study and was granted under a priority review process. The study was an open-label, single-arm trial evaluating safety and pharmacokinetics in children younger than 8 years with stage 2 T1D. A total of 23 patients enrolled and received intravenous teplizumab once daily for 14 consecutive days, with follow-up extending up to 26 months.

T1D is a progressive autoimmune disease characterized by the destruction of insulin-producing beta cells. Stage 2 disease is defined by the presence of at least two diabetes-related autoantibodies and dysglycemia due to progressive loss of beta-cell function. Stage 3 T1D is associated with hyperglycemia and requires insulin therapy and may present with symptoms such as polyuria, polydipsia, weight loss, and fatigue.

Teplizumab is a CD3-directed monoclonal. The agent previously received breakthrough therapy and orphan drug designations in the US. 

The drug is already approved in multiple regions, including the EU, UK, China, Canada, and others, to delay the onset of stage 3 T1D in patients aged 8 years and older with stage 2 T1D. Regulatory review is ongoing for additional indications, including use in patients recently diagnosed with stage 3 disease.

Source: Sanofi