Sanofi announces Tzield sBLA accepted for expedited review by FDA

The US Food and Drug Administration has accepted for expedited review the supplemental biologics license application for Tzield to delay the progression of stage 3 type 1 diabetes in adults and pediatric patients eight years of age and older recently diagnosed with stage 3 T1D. The FDA nominated Tzield for the Commissioner’s National Priority Voucher pilot program based on its potential to address a large unmet medical need. The CNPV program aims to shorten the review process from what normally takes 10-12 months to 1-2 months, while maintaining FDA’s rigorous safety and efficacy standards. The sBLA is supported by the results from the PROTECT phase 3 study, which met its primary endpoint, evaluating preservation of beta cell function as assessed by significantly slowing the decrease in mean C-peptide levels at trial study completion, compared to placebo. Additionally, the sBLA builds on the clinical development program of Tzield including approximately~1,000 patients. Adverse events observed in the PROTECT phase 3 study were consistent with previous studies. Most common adverse events were headache, nausea, rash, lymphopenia, leukopenia and gastrointestinal symptoms, consistent with the mode of action of cytokine release. 1.8% of those who received Tzield in the PROTECT study developed cytokine release syndrome possibly or probably related to Tzield. Additionally, Tzield is being reviewed under the accelerated approval program, a pathway that allows the FDA to review therapies intended to treat serious conditions that fill an unmet medical need, based on a surrogate endpoint reasonably likely to predict clinical benefit. In line with this requirement, the confirmatory BETA-PRESERVE phase 3 study was initiated recently and is currently enrolling participants.