Diabetes Care perspective, developed by experts across the country and endorsed by Breakthrough T1D and the Pediatric Endocrine Society, helps pediatric diabetes teams integrate teplizumab into care for recently diagnosed children ages 8 to 17
For the first time, children ages 8 to 17 who are newly diagnosed with type 1 diabetes have access to an approved treatment that targets the underlying autoimmune disease, rather than solely managing blood sugar levels with insulin.
A national group of clinical trial investigators and pediatric diabetes specialists, led by a physician-scientists at the Barbara Davis Center for Diabetes at theUniversity of Colorado Anschutz, has published practical guidance to help care teams and families navigate this new option.
The treatment, teplizumab, is an immunotherapy that slows the immune system's destruction of the body's remaining insulin-producing cells. In June 2026, the U.S. Food and Drug Administration (FDA) expanded its approval to include children and adolescents ages 8 to 17 years recently diagnosed with stage 3 type 1 diabetes (the stage when symptoms appear and insulin is needed), to delay the decline in their own insulin production. This new indication was granted under the FDA's accelerated approval pathway, based on the treatment's effect on preserving insulin production. Teplizumab was already approved to delay the onset of stage 3 type 1 diabetes in people ages 1 and older with stage 2 type 1 diabetes.
Kimber Simmons, MD, MS, an associate professor of pediatrics at the University of Colorado Anschutz School of Medicine and a physician-scientist at the Barbara Davis Center for Diabetes, led the development of the Diabetes Care perspective with a national group of co-authors and support from Breakthrough T1D. Drawing on complementary expertise in clinical trials and real-world care, the team addresses how to identify appropriate candidates, discuss potential benefits and risks with families and integrate treatment into comprehensive diabetes care. Simmons co-authored the published results of both PROTECT, the trial in newly diagnosed children and adolescents that supported the expanded approval, and PETITE-T1D, a study of teplizumab in children younger than 8 with stage 2 type 1 diabetes.
"Insulin is essential in stage 3 type 1 diabetes, but it doesn't address the immune process that is destroying the cells that make insulin," said Simmons. "Teplizumab gives us an opportunity to preserve some of the beta cells and insulin-producing capacity that a person still has. The goal is to intervene early enough to make a meaningful difference."
How Teplizumab Works
Type 1 diabetes is an autoimmune disease in which the immune system attacks the beta cells in the pancreas that make insulin, a hormone needed for the body to use energy from food and to regulate blood sugar. By the time a child is diagnosed with stage 3 type 1 diabetes, symptoms have appeared, but some insulin-producing cells usually remain.
Teplizumab targets the immune cells involved in destroying beta cells. The goal is to preserve some of the body's own insulin production and possibly extend the "honeymoon" period after diagnosis, when a person still makes some of their own insulin.
In the PROTECT trial of 328 children and adolescents, those who received teplizumab kept significantly more insulin-producing capacity after 78 weeks, measured by C-peptide (a marker of the body's own insulin production), than those who received placebo. Nearly 95% of children treated with teplizumab maintained a clinically meaningful level of their own insulin production, compared with about 79% of those given placebo. In separate studies of people with type 1 diabetes, higher C-peptide levels have been linked with fewer episodes of severe low blood sugar, lower insulin needs and a lower risk of some complications, such as diabetic eye disease.
Treatment is given as two 12-day courses of daily IV infusions, usually six months apart. Potential side effects include rash, headache, nausea and a temporary drop in lymphocytes, a type of white blood cell. More serious risks include cytokine release syndrome (an inflammatory reaction), infections and viral reactivation. For this reason, treatment requires an experienced team and careful monitoring.
Teplizumab does not cure type 1 diabetes or replace insulin treatment. It adds a way to target the underlying immune process while standard diabetes care continues.
Why Timing Matters
Treatment must begin within eight weeks of diagnosis, so families have only a few weeks to learn about the option and decide if they want treatment. In the PROTECT clinical trial that supported the approval, children and adolescents enrolled within six weeks of diagnosis. Earlier studies suggested that treatment soon after diagnosis produces stronger responses, a short window emphasizing the need for guidance.
Bringing Teplizumab into Everyday Care
"Once a treatment is approved, the question becomes: How do we actually do this in the clinic?" said Simmons, who also practices at Children's Hospital Colorado. "The way patients are treated in the real world doesn't always line up perfectly with a clinical trial protocol. We wanted to give clinicians practical guidance so they can have informed conversations with families and make thoughtful, safe decisions."
"Research shows that, on average, it takes 17 years for landmark medical breakthroughs to become widely adopted in routine clinical practice. Closing that gap is essential to getting the best science to people sooner," said Breakthrough T1D Senior Vice President of Medical Affairs Anastasia Albanese-O'Neill, Ph.D., APRN, CDCES. "Breakthrough T1D is focused on efforts that will close this gap in a way that is safe and supports shared decision making between clinicians and families. This new clinical guidance serves as an important tool that will help guide timely care for children with a new stage 3 type 1 diabetes diagnosis. We're grateful to the leaders who provided their expertise and time to develop it."
Combining clinical trial evidence and experience across care settings, the authors offer a framework for patient selection, shared decision-making, safety monitoring and treatment planning. A central message is that teplizumab should be coordinated with established diabetes care rather than treated as a separate service.
"We don't want to create a separate pathway that fragments care," said Simmons. "This is an opportunity to be thoughtful about how we use a new therapy while keeping the whole patient in view. Our specialty needs to stay focused on providing the best coordinated care we can."
Key Points:
- First treatment of its kind for newly diagnosed children: Teplizumab is now approved, under accelerated approval, to slow the loss of the body's own insulin production in children ages 8 to 17 recently diagnosed with stage 3 type 1 diabetes. It is also approved to delay the onset of stage 3 disease in people ages 1 and older with stage 2 type 1 diabetes.
- Timing matters: Treatment should start as soon as possible after diagnosis and no later than eight weeks after, making practical guidance critical.
- National collaboration led from Colorado: A CU Anschutz-led team of clinical trial investigators and clinicians from across the country developed the Diabetes Care perspective with support from Breakthrough T1D.
- Benefits and risks: In a clinical trial, nearly 95% of treated children kept meaningful insulin production at 78 weeks, compared with about 79% on placebo. Extended follow-up is ongoing, and a confirmatory trial is underway. Treatment requires IV infusions and monitoring for side effects.
- Insulin remains essential: Teplizumab does not cure type 1 diabetes or replace insulin and ongoing diabetes care.
