Sanofi (SNY) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for Tzield (teplizumab-mzwv), intended to delay the decline of endogenous insulin production in children aged 8 to 17 who have been recently diagnosed with stage 3 type 1 diabetes (T1D). The therapy is not indicated for use as a disease-modifying treatment in non-autoimmune dysglycemic conditions.
The FDA’s decision was supported by results from the phase 3 PROTECT study, alongside data from a comprehensive global clinical development program involving more than 900 patients treated with the drug.
Reported adverse reactions include lymphopenia, vomiting, rash, leukopenia, diarrhea, neutropenia, increased liver transaminase, and headache. More severe risks include cytokine release syndrome and life-threatening viral reactivation; consequently, immunocompromised patients are at a heightened risk for viral reactivation.
This accelerated approval is based on evidence showing a reduction in C-peptide decline. Maintenance of this approval may depend on further verification of clinical benefits through confirmatory studies.
Tzield has previously received approval to delay the onset of stage 3 T1D in adults and children aged eight and older with stage 2 T1D. It is also authorized for the same indication in several global markets, including the UK, the EU (marketed as Teizeild), China, Australia, Canada, Israel, Saudi Arabia, the UAE, Kuwait, Brazil, and Switzerland. Regulatory reviews continue in additional jurisdictions.