Better education on screening required for widespread use of teplizumab in type 1 diabetes treatment

Teplizumab (Tzield, Sanofi) may delay the onset of type 1 diabetes (T1D), but barriers to treatment may affect adherence to the medication. Increased screening opportunities and education about these programs can help close the treatment gap for initiating teplizumab. Pharmacists should be aware of and stay up to date on these programs for their patients.

T1D is a chronic autoimmune disease that causes hyperglycemia, but the underlying cause of T1D development has not been identified. However, there is enough knowledge about the disease to prevent hyperglycemia and clinical diagnosis is available, and efforts are being made to focus on beta cell autoimmunity in patients with high genetic risk. In addition, another preventative approach is to protect the functional beta cells that are still present. Islet autoantibodies (IA) are currently used as a standard immune biomarker to determine the risk of T1D, according to the authors of a review published in 2010. Pharmacology Research.¹

There are various stages of prevention: primary, secondary, and tertiary. For primary prevention, the researchers noted that it is difficult to intervene before the onset of IA. Therefore, primary prevention is often targeted at direct relatives with high genetic risk of human leukocyte antigen class II genes. Secondary prevention is often targeted at individuals in whom IA has been detected and focuses on treatments such as parenteral insulin, oral insulin, and glutamic acid decarboxylase. The authors reported that immune interventions are being studied to slow the decline of beta cell function, with teplizumab being the most notable agent.¹

According to the review authors, teplizumab has been shown to delay the onset of stage 3 T1D and is considered a milestone in the field of T1D because it is the first immunomodulatory agent to delay the onset of T1D in adults and high-risk children aged 8 years and older.

The FDA approved the drug, a CD3 antibody, in November 2022. In phase 2 trials, teplizumab preserved insulin production and reduced exogenous insulin, a review published in November 2020 reported. Touch Reviews in EndocrinologyIn a phase 3 trial, the drug demonstrated the ability to reduce the decline in mean area under the curve of C-peptide after 2 years. Approximately 77% of patients receiving teplizumab developed anti-drug antibodies.^1,2 The phase 3 PROTECT trial (NCT03875729) ended in May 2024, and the TrialNet 10 extension trial (NCT04270942) ended in January 2024. Both were used to further explore the effects of the drug.^3,4

Healthcare professionals should consider using teplizumab in select patients because it can delay the clinical diagnosis of T1D by 2–3 years. This recommendation is from the 2024 American Diabetes Association Standards of Care. However, the cost is a major barrier: $13,850 per vial, or approximately $194,000 for 14 days of treatment. To be eligible for treatment, patients must be screened for T1D IA when asymptomatic, and previous guidelines recommended screening for autoantibodies as part of a research study or for first-degree family members of T1D patients.^Five

In a review published in DiabetesThe authors note that approximately 90% of patients who develop type 1 diabetes have no family history, highlighting the need for screening opportunities in the general population. According to the Breakthrough T1D website, there are three programs designed for early detection of type 1 diabetes: TrialNet, Autoimmune Screening for Children (ASK), and Population-Level Estimation of Type 1 Diabetes Risk Genes in Children (PLEDGE).^6,7

TrialNet is a US early detection and clinical trial program targeting individuals aged 2.5-45 years with a first-degree relative with T1D, 2.5-20 years with a second-degree relative with T1D, or 2.5-45 years who have tested positive for at least one T1D-related IA outside of a clinical trial. ASK includes individuals aged 1-17 years in the US without a family condition, and PLEDGE includes children under the age of 6 receiving care at Stanford Health in South Dakota.⁷

Teplizumab has the potential to significantly transform patients' lives by delaying the onset of type 1 diabetes. However, screening for type 1 diabetes IA before symptoms appear is required, and educating patients about these screening opportunities is important in order to receive treatment with teplizumab.⁶