The National Institute for Health and Care Excellence (Nice) has approved teplizumab, a novel therapy designed to delay the onset of type 1 diabetes by up to three years. This decision marks a significant advancement, with Diabetes UK calling it the beginning of a "new age of treatment."
Teplizumab, marketed as Tzield by Sanofi, is indicated for children aged eight and above, as well as adults in the early stages of the condition before symptoms manifest. Type 1 diabetes affects approximately 400,000 people in the UK, a chronic condition where the immune system mistakenly attacks the insulin-producing cells in the pancreas. Teplizumab works by modulating the immune system to prevent this attack.
Clinical evidence suggests that this one-off course of treatment can postpone the symptomatic onset of type 1 diabetes, allowing individuals, particularly children, more time before intensive diabetes management becomes necessary. Nice estimates that around 1,100 people may be eligible for the drug in its first year of availability, with projections suggesting 820 eligible individuals in subsequent years.
Dr. Elizabeth Robertson of Diabetes UK highlighted the drug's potential to shift treatment paradigms. "For the first time in 100 years, we are moving beyond insulin with a medicine that targets the root cause of the condition," she stated, adding that this development "represents a shift towards a future where type 1 diabetes can be prevented altogether." She emphasized that the drug offers individuals in the early stages extra years free from the daily demands of insulin management and stressed the importance of early detection to maximize these benefits.
Two studies are currently underway in the UK to identify individuals at risk of type 1 diabetes: the Early Surveillance for Autoimmune Diabetes study, focusing on children aged two to 17, and the T1DRA study, screening adults aged 18 to 70. Early identification is crucial for enabling eligible patients to receive teplizumab.
Helen Knight, director of medicines evaluation at Nice, described the recommendation as "genuinely exciting," noting that evidence indicates an average delay of nearly three years in the symptomatic onset of diabetes. Karen Addington of Breakthrough T1D echoed this sentiment, calling it an "incredible moment."
Teplizumab is administered intravenously over 14 consecutive days. Each infusion lasts approximately 30 minutes, with the dosage gradually increasing over the initial days of treatment.