Sanofi Withdraws Diabetes Drug Teplizumab from FDA Fast-Track Review Amid Safety Concerns
Key Insights
Sanofi has requested to withdraw its diabetes drug teplizumab (Tzield) from the FDA's new fast-track voucher program following disagreements over safety concerns.
FDA Center Director Tracy Beth Høeg opposed staff approval recommendations, citing concerns that the drug's benefits do not outweigh risks including Epstein-Barr virus (search) and cancer (search) development.
The drug was under review to delay progression of stage 3 type 1 diabetes (search) in patients eight years and older, having already received approval for patients as young as one year.
Sanofi has asked the U.S. Food and Drug Administration to remove its diabetes drug teplizumab from the regulator's new fast-track review program, following internal disagreements over the medication's safety profile. The pharmaceutical company's decision comes after Tracy Beth Høeg, director of the FDA's Center for Drug Evaluation and Research, opposed a staff recommendation to approve the drug for an expanded indication.
Regulatory Dispute Over Safety Profile
The controversy centers on teplizumab, marketed as Tzield, which was being reviewed to delay the progression of stage 3 type 1 diabetes (search) in patients eight years of age and older. The drug received approval in the U.S. last month for delaying the onset of stage 3 T1D in patients as young as one year and older.
According to sources familiar with the dispute, Høeg expressed concerns that the drug's benefits do not outweigh its risks, particularly regarding the potential for developing Epstein-Barr virus (search) and cancer (search). "We will not comment on our confidential ongoing discussions with the FDA regarding our supplemental biologics application... which is currently pending a decision," a Sanofi spokesperson stated.
Safety Data Under Scrutiny
Sanofi has reported observing three cancer (search) cases among more than 1,000 patients treated with teplizumab over a 30-year period. The company maintains that no causal relationship was established between the cancers and teplizumab treatment, noting that no cases were reported in post-marketing surveillance.
The current safety concerns follow previous regulatory challenges for Tzield. In January, the FDA stalled its review of the drug over adverse events, including two incidents related to seizure and blood clotting, and one death.
Impact on Fast-Track Program
The withdrawal request affects Sanofi's participation in the FDA commissioner's national priority voucher program, which was launched last year. This program promises regulatory decisions within one to two months, compared to the typical timeline of 10-12 months, for a limited number of drugs deemed critical to public health or national security.
The dispute highlights the ongoing challenges in balancing therapeutic benefits against potential risks in diabetes treatment, particularly for medications targeting the progression of type 1 diabetes (search) in pediatric and young adult populations.