Tzield, first drug to delay type 1 diabetes onset, receives FDA approval

USA: Tzield (teplizumab-mzwv), an injection that delays the onset of stage 3 type 1 diabetes (T1D), has received approval from the U.S. Food and Drug Administration (US FDA). The injection delays the onset in adults and pediatric patients eight years and above who currently have stage 2 type 1 diabetes.

In the clinical trial, TZIELD delayed the median onset of Stage 3 T1D by 25 months or approximately two years in patients with stage 2 T1D, compared to placebo. Stage 3 T1D is linked with significant health risks, including diabetic ketoacidosis, which can be life-threatening. In due course, patients who progress to Stage 3 T1D need insulin injections for life.

Type 1 diabetes is a disease that occurs when the immune system attacks and destroys the cells that produce insulin. Individuals with the diagnosis of type 1 diabetes have increased blood sugar levels that require insulin to survive and control their blood sugar levels. Although the condition can affect people of any age, it is usually diagnosed in children and young adults. A parent, brother or sister with type 1 diabetes raises a person's risk for T1D, although most T1D patients do not have a family history.

Tzield binds to specific immune system cells and delays progression to stage 3 type 1 diabetes. It may deactivate the immune cells that attack insulin-producing cells and increase the proportion of cells that help moderate the immune response. The administration of Tzield is via intravenous (IV) infusion once daily for 14 consecutive days.

"Today's approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients," John Sharretts, M.D., director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA's Center for Drug Evaluation and Research said in a press release. "The drug's potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease."

The approval is based on evaluating Tzield's safety and efficacy in a randomized, double-blind, event-driven, placebo-controlled trial. It included 76 patients with stage 2 type 1 diabetes. The primary efficacy measure was the time from randomization to the development of stage 3 type 1 diabetes diagnosis. Patients were randomly allocated to Tzield or a placebo once daily via IV infusion for 14 days. The patients were followed for a median of 51 months.

Based on the study, the authors reported the following:

• 45% of the 44 patients who received Tzield were later diagnosed with stage 3 type 1 diabetes, compared to 72% of the 32 patients who received a placebo.
• The mid-range time from randomization to stage 3 type 1 diabetes diagnosis was 50 months for the patients who received Tzield and 25 months for those who received a placebo. This represents a statistically significant delay in developing stage 3 type 1 diabetes.

The most common side effects of Tzield include rash, headache, and decreased levels of specific white blood cells. Tzield use comes with warnings and precautions, including premedicating and monitoring for symptoms of Cytokine Release Syndrome; reduced levels of a type of white blood cell called lymphocytes; risk of hypersensitivity reactions; risk of serious infections; the need to administer all age-appropriate vaccinations before starting Tzield; as well as avoiding concurrent use of live, inactivated and mRNA vaccines with Tzield.