Key Takeaways:
The FDA granted accelerated approval to Sanofi's Tzield for children aged 8 to 17 with newly diagnosed stage 3 type 1 diabetes, the first disease-modifying therapy for this group.
"We now have a novel therapy that targets the autoimmune and progressive nature of stage 3 type 1 diabetes," Aaron J. Kowalski, chief executive officer of Breakthrough T1D, said in a statement.
The decision, announced June 12, was supported by the Phase 3 PROTECT trial, which randomized 328 children and adolescents 2-to-1 to Tzield or placebo. Patients received two 12-infusion courses six months apart. Tzield slowed the decline in mean C-peptide area under the curve by 0.13 pmol/mL compared with placebo, as measured through a four-hour mixed-meal tolerance test. The most common adverse events included lymphopenia, vomiting, rash and headache, while serious events such as cytokine release syndrome have been reported.
The expansion opens Tzield to patients diagnosed at stage 3, when symptoms first appear — a far larger population than the stage 2 patients previously eligible. Approximately 64,000 Americans are diagnosed with type 1 diabetes each year, according to Breakthrough T1D. Sanofi acquired the drug through its $2.9 billion purchase of Provention Bio in 2023.
Tzield was first approved in November 2022 to delay the onset of stage 3 disease in patients with stage 2 type 1 diabetes. The FDA expanded that label in April 2024 to include children as young as one year old. Friday's accelerated approval extends the drug's use to preserving endogenous insulin production after diagnosis.
The approval comes after internal disagreement at the FDA over the drug's risk-benefit profile. Tracy Beth Høeg, then acting director of the Center for Drug Evaluation and Research, reportedly opposed the expansion, arguing that Tzield's benefits did not outweigh its risks in this population, according to STAT News. Sanofi had requested to withdraw Tzield from the Commissioner's National Priority Voucher program in May after Høeg's involvement. Høeg was fired later that month and has been succeeded by CDER Deputy Director Michael Davis on an acting basis.
Sanofi is required to confirm clinical benefit through the Phase 3 BETA-PRESERVE trial, which is currently enrolling and expected to read out in 2028. The company did not disclose updated sales projections for the expanded label.
The label expansion gives Sanofi access to a significantly larger addressable patient population, as most type 1 diabetes diagnoses occur at stage 3 when symptoms emerge. Investors will watch enrollment in the BETA-PRESERVE confirmatory trial and any pricing updates for the expanded indication.
This article is for informational purposes only and does not constitute investment advice.
