The immunotherapy, teplizumab shown to delay the development of type 1 diabetes has been given the green light by an official regulatory board in the United States

Abstract

The U.S. Food and Drug Administration (FDA) has recently approved a first-in-class therapy, teplizumab to delay the onset of type 1 diabetes in adults and children aged eight years and older. Teplizumab is an immunotherapy which has been shown to deactivate the immune cells that attack insulin-producing cells, while increasing proportion of cells that help moderate the immune response. Teplizumab is administered by intravenous infusion once daily for 14 consecutive days. This approval adds an important new treatment options for at –risk people to delay clinical diagnosis of type 1 diabetes and may provide people with months to years without the burdens of disease. This may improve clinical outcome given the well recognised association between “life-time disease burden” with risks of long-term vascular complications. The safety and efficacy of Teplizumab were previously evaluated in a randomized, double-blind, event-driven, placebo-controlled trial with 76 people with stage 2 type 1 diabetes. In the trial, participants randomly received teplizumab or a placebo once daily via intravenous infusion for 14 days. The primary measure of efficacy was the time from randomization to development of stage 3 type 1 diabetes diagnosis. The trial showed that over a median follow-up of 51 months, 45% of the 44 people who received teplizumab were later diagnosed with stage 3 type 1 diabetes, compared to 72% of the 32 patients who received a placebo. The mean time from randomization to stage 3 type 1 diabetes diagnosis was 50 months for those who received teplizumab and 25 months for those who received a placebo. This represents a statistically significant delay in the development of stage 3 type 1 diabetes, which would reduce the time to start intensive therapy to manage hyperglycaemia. Crucially, Diabetes UK has suggested that preventative treatments for type 1 diabetes can only be effective when combined with screening programmes to identify those at risk. It is anticipated that this approval will open the door for increased research investment, to develop further effective immunotherapies to prevent the development of type 1 diabetes.