FDA approves intravenous injection for delayed onset of stage 3 type 1 diabetes

The FDA approved teplizumab-mzwv (Tzield ; Provention Bio) intravenous injection to delay the onset of stage 3 type 1 diabetes in adults and children 8 years and older with stage 2 type 1 diabetes, the FDA announced on November 17. This is the first drug approved for this indication.

John Sharretts, MD, director of the Division of Diabetes, Lipid Disorders, and Obesity of the FDA’s Center for Drug Evaluation and Research, sees this as an important option for certain at-risk patients. “The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease,” he stated.

Teplizumab may work by binding to certain immune system cells that attack insulin-producing cells and increase the proportion of cells that help moderate the immune response, thus delaying progression to stage 3 type 1 diabetes.

Clinical trial of teplizumab

The FDA approval of teplizumab was based on the results of a randomized, double-blind, event-driven, placebo-controlled trial of safety and efficacy that included 76 patients with stage 2 type 1 diabetes.

The patients were randomized to teplizumab (44 patients) or placebo (32 patients). The primary efficacy outcome was the time from randomization to development of stage 3 type 1 diabetes. Patients underwent an intravenous injection once daily for 14 consecutive days.

The investigators reported that 45% of the patients randomized to teplizumab were diagnosed later with stage 3 type 1 diabetes compared with 72% of the patients randomized to placebo during a median follow-up of 51 months.

The results showed that the mid-range time from randomization to the diagnosis of stage 3 type 1 diabetes was 50 months for the patients who received teplizumab and 25 months for those randomized to placebo, a difference in the time to onset of stage 3 type 1 diabetes that reached significance.

Decreases in the levels of lymphocytes, rashes, and headaches were the most side effects of teplizumab. In addition, patients should be monitored for symptoms of cytokine release syndrome, decreased lymphocyte levels, and the risk of serious infections and hypersensitivity reactions. Physicians are advised that patients should receive all age-appropriate vaccinations before teplizumab is started and avoid concurrent use of live, inactivated and mRNA vaccines.

Teplizumab received Priority Review and Breakthrough Therapy designations for this indication.