Sanofi has asked the United States Food and Drug Administration to remove its type 1 diabetes drug, teplizumab (Tzield), from the Commissioner’s National Priority Review program following internal disagreement about expanding its use, according to a May 7 report.
The move is significant because it comes amid debate within the FDA about whether to approve Tzield for a broader patient group. FDA staff had supported expanding Tzield’s label to include adults and children ages eight and older with stage 3 type 1 diabetes, but acting Center for Drug Evaluation and Research Director Tracy Beth Høeg disagreed, citing concerns that the benefits may not outweigh potential risks.
Tzield was previously approved as the first drug to delay onset of stage 3 type 1 diabetes in adults and children with stage 2 disease. Last month, an additional indication was added for delaying onset in children as young as one year old. Under the FDA's CNPV program—which aims to shorten review times—Sanofi expected a decision by April 21. However, this timeline was delayed due to safety concerns including seizures, blood clotting events, and one death. Høeg is reportedly especially concerned about Epstein-Barr virus reactivation and cancer risk; Tzield’s label warns that “serious, life-threatening cases of viral reactivation, including Epstein-Barr virus (EBV) and cytomegalovirus (CMV) reactivation have been reported with TZIELD.”
A Sanofi spokesperson declined to comment on ongoing discussions with the FDA but confirmed that its supplemental biologics application for Tzield remains pending regulatory decision. The spokesperson said: “Over 1,000 patients have been treated with teplizumab over the course of 30 years of clinical development,” adding that three malignancy cases were observed but no causal link established between them and teplizumab or in post-marketing surveillance. On reports of one death associated with Tzield: “Based on all available information, no causal relationship has been established between Tzield and the single case with a reported fatal outcome.”
FDA Commissioner Marty Makary told CNBC this week that such interventions by political appointees do not occur at his agency: “Every ‘accept’ or ‘reject’ decision at the FDA, on my watch, has been the accept or reject recommendation of the primary review teams at the agency.” Despite Makary’s statement, there have been other recent instances where senior leaders intervened in drug reviews during his tenure.
As of publication time, BioSpace reports that it has not received confirmation from FDA regarding Sanofi’s request for removal from priority review.
