FDA Approves Tzield for Stage 3 Type 1 Diabetes; Canadian Pediatric First Reported

Tzield (teplizumab), the first disease-modifying therapy approved by the FDA to delay progression of type 1 diabetes (T1D), has now been approved by the FDA for use in stage 3 T1D, and has been administered to children in Canada for the first time. People aged 8–17 with stage 3 T1D will soon be able to get Tzield in clinics if they have been diagnosed within the last 8 weeks. This makes it the first disease-modifying therapy approved for individuals in stage 3 T1D. Further promising news out of the United States is that the FDA has approved Tzield for children aged one year and above with stage 2 T1D.

The FDA reviewed data from the PROTECT clinical trial to support Tzield's approval. The PROTECT study demonstrated that Tzield could safely and effectively preserve insulin-producing beta cells in newly diagnosed children and adolescents with stage 3 T1D. Clinical data shows that individuals with more beta cell function also have better diabetes-related outcomes, like improved HbA1c, more time in range, less hypoglycemia, and less insulin use. Tzield was approved through FDA's accelerated approval pathway, and it is the first time a disease-modifying therapy has been granted approval based on C-peptide, a marker of beta cell function.

Tzield is designed to delay progression of T1D in people who show early signs of the disease but do not yet need insulin. Clinical studies show it can delay the onset of insulin-dependent diabetes by a median of about two years in eligible adults and children. Tzield binds to specific receptors on immune cells, disabling them from destroying insulin-producing beta cells in the pancreas. It is administered as a daily intravenous infusion for 14 days.

In Canada, Tzield has been administered to children for the first time – two patients at BC Children's Hospital with early-stage type 1 diabetes. The medication teplizumab was provided on a compassionate basis by Sanofi as part of a research-clinical collaboration. A small number of adults have already received the drug since it was approved by Health Canada in 2025. In Canada, Tzield is currently approved for age 8 and above. Although approved as a therapy by Health Canada, in January Canada's Drug Agency has announced a recommendation not to reimburse Tzield.

To be eligible for Tzield, one has to be identified through screening as having early stage T1D. Screening for T1D identifies the presence of certain autoantibodies, which can mean someone is at high risk of developing type 1 diabetes. In Canada, family members of someone with type 1 diabetes can be screened through TrialNet (for first- and second-degree relatives anywhere in Canada) and by FEDERATE-Can (for first-degree relatives in Quebec). However, only 10-15% of newly diagnosed individuals have a family history, so familial screening alone will miss over 85% of new cases.

UC Davis Health now offers Tzield, a first-in-class biologic therapy designed to delay the onset and progression of T1D. It is the only healthcare system in the Sacramento region and inland northern California to offer this T1D therapy. This prescription monoclonal antibody is available for adults and children 1 year of age and older with stage 2 T1D, in which patients are pre-symptomatic, but blood glucose becomes irregular, and stage 3 T1D, in which patients are recently diagnosed within eight weeks. UC Davis Health offers screening to first-degree relatives of individuals with type 1 diabetes because they are at increased risk of developing this condition.

Researchers at the University of Missouri School of Medicine have developed a method to transplant insulin-producing islets for T1D that may eliminate the need for immunosuppressive drugs. The approach, published in JCI Insight, uses ex vivo engineering of donor islets with immune-regulating molecules that reduce both innate and adaptive immune rejection after transplantation. By adding thrombomodulin (TM) and CD47 to the surface of islets, the team created a localized immune-modulating barrier that allowed transplanted cells to survive and function while still responding to glucose and producing insulin. In allogenic mouse models, the grafts survived from 120 to 330 days in eight of the 11 mice tested without immunosuppression; by comparison, unmodified islets were rejected quickly with survival time averaging 12 days. Normal blood glucose levels were achieved in more than 72% of the mice. The researchers said that additional work now needs to evaluate the safety and effectiveness of these cells in humans.

A small clinical trial into a stem-cell based treatment for T1D at UCLA has been successful enough that the trial expanded to include 50 participants. The most recent updates indicate that the first participant's body continues to produce insulin, but the person is also required to take immunosuppressive drugs to keep his body from rejecting the islet cells that are producing insulin.

UW Medicine is helping to conduct a clinical trial exploring an investigational therapy for people diagnosed with type 1 diabetes. Adults ages 18 to 35 with a recent diagnosis are prospective candidates to test a drug designed to protect the insulin-producing cells in the pancreas. By preserving the body's ability to make its own insulin, the investigational therapy, which is delivered via IV, might reduce long-term effects of the disease.

A review in JCI notes that teplizumab was shown to attenuate loss of beta cell function that occurs over time and delay progression to clinical disease in individuals at risk, leading to its regulatory approval in 2022. Despite major improvements in insulin pharmacokinetics, algorithms for automated insulin delivery, and continuous glucose monitoring, fewer than one quarter of individuals with T1D achieve the recommended glycemic targets. Future studies may involve combinations of agents to extend immunologic tolerance and protect and restore beta cells so that lasting metabolic remission can be achieved.