mRNA-1010

iStock, Khafizh Amrullah Acting Center for Drug Evaluation and Research Director Tracy Beth Høeg reportedly disagreed with staff who wanted to approve Sanofi’s type 1 diabetes drug. It’s far from the first time a political appointee has allegedly meddled in a recent FDA decision. Sanofi has reportedly requested that the FDA remove teplizumab, or Tzield, from its Commissioner’s National Priority Review program after a high-level disagreement over whether to approve the type 1 diabetes drug. FDA staff had been in favor of expanding Tzield’s label to include adults and kids ages 8 years and older with stage 3 type 1 diabetes, according to STAT News on Wednesday, citing anonymous sources. But acting Center for Drug Evaluation and Research Director Tracy Beth Høeg disagreed, raising concerns that the drug’s benefits do not outweigh the risks. Tzield is the first drug approved to delay the onset of stage 3 type 1 diabetes in adults, as well as an option for children 8 years and older, with stage 2 disease. Sanofi added another indication to Tzield’s label last month when the FDA approved the drug to delay the onset of stage 3 type 1 diabetes in children as young as one-year old. Stage 3 type 1 diabetes occurs when the body cannot make enough insulin on its own and insulin injections may be required. Under the FDA’s Commissioner’s National Priority Review (CNPV) program, introduced last June, Sanofi had expected to receive a decision regarding the new indication by April 21. The agency pushed back the date in January, however, after detecting concerning safety signals, including two seizures, one episode of blood clotting and one death, Reuters reported at the time. Now, Høeg is said to be especially concerned about Epstein-Barr virus and cancer risk, according to STAT . The drug’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.” The CNPV program is intended to shorten review times from 10–12 months to 1–2 months for investigational products that align with certain national priorities. Sanofi, which did not apply for the program, was granted a CNPV for Tzield for treating stage 3 diabetes in October 2025. A Sanofi spokesperson declined to clarify the current regulatory status of Tzield, telling BioSpace in an email Wednesday afternoon that the company “will not comment on our confidential ongoing discussions with the FDA.” The company did confirm that its supplemental biologics application (sBLA) for Tzield is currently pending a regulatory decision. In the same email, Sanofi stood behind that application. “Over 1,000 patients have been treated with teplizumab over the course of 30 years of clinical development,” the spokesperson said, adding that three cases of malignancies have been observed in these programs but that in each case, “no causal relationship was established between the malignancies and teplizumab” and “zero cases have been reported in post-marketing surveillance.” The French pharma also responded to the reports of one death associated with the drug. “Based on all available information, no causal relationship has been established between Tzield and the single case with a reported fatal outcome,” the spokesperson said. As of publication, the FDA has not responded to BioSpace’s request for confirmation of Sanofi’s reported request to remove Tzield from the CNPV program. A common refrain This report comes on the heels of a CNBC interview on Tuesday during which FDA Commissioner Marty Makary claimed that this type of intervention from a political appointee does not happen at the 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,” Makary told CNBC . Despite Makary’s assertion, however, the apparent intervention in Tzield’s review is far from the first by a senior FDA leader during his tenure. Longtime oncology chief Richard Pazdur reportedly had a hand in issuing the first rejection of Replimune’s advanced melanoma drug, RP1, last July. The FDA did not consider RP1’s registrational trial to be an “adequate and well-controlled clinical investigation,” according to Replimune’s press release at the time. The agency again turned away RP1 last month. Immuno-oncology Top FDA Cancer Regulator Intervened To Reject Replimune’s Melanoma Drug According to reporting from multiple outlets, Richard Pazdur, head of the Oncology Center of Excellence at CDER, opposed the consensus opinion of CBER staff to approve the drug. Replimune’s stock has dropped precipitously since the rejection. August 4, 2025 · 2 min read · Dan Samorodnitsky Read more Analysts had originally speculated that it was former Center for Biologics Evaluation and Research (CBER) director Vinay Prasad who was responsible for the first Replimune CRL. While it turned out to be Pazdur in that case, Prasad—who departed the FDA last week after a tumultuous one-year tenure —did reportedly have a hand in the rebuff of Capricor Therapeutics’ cell therapy deramiocel for Duchenne muscular dystrophy cardiomyopathy, also in July last year. That rejection, which was as a surprise to Capricor CEO Linda Marbán, came after Prasad allegedly canceled an advisory committee meeting that had been scheduled to discuss the treatment. Speaking with BioSpace immediately following Prasad’s first, short-lived exit from the FDA in July 2025 after a spat with Sarepta, Marbán said , “We all understand the ramifications of [Prasad’s return], that there’s not a lot of ladders you can go up besides the Prasad ladder.” Duchenne muscular dystrophy With Prasad Back at FDA, Capricor To Fight DMD Cardiomyopathy Rejection Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset. August 20, 2025 · 7 min read · Heather McKenzie Read more Then, in February, chaos broke out when the FDA refused to review Moderna’s investigational flu vaccine, mRNA-1010. In the refusal-to- signed by Prasad and published in full on the company’s website, the agency claimed that Moderna failed to support mRNA-1010’s application with an “adequate and well-controlled” trial. The FDA reversed course the following week, promising Moderna an August decision date on an amended filing for the candidate. As for Sanofi’s Tzield, Høeg has called for an adcomm after it became apparent she would need to override FDA staff, according to STAT on Wednesday. No such meeting has been scheduled.