FDA Approves Teplizumab-mzwv, the First Drug to Delay Onset of Type 1 Diabetes

The FDA approved teplizumab-mzwv (Tzield, Provention Bio) injection to delay the onset of stage 3 type 1 diabetes in adults and pediatric patients aged 8 years and older who currently have stage 2 type 1 diabetes.

“Today’s approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients,” said John Sharretts, MD, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research, 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.”

Type 1 diabetes occurs when the immune system attacks and destroys the cells that make insulin, and people who have been diagnosed with type 1 diabetes have increased glucose that requires insulin shots, or wearing an insulin pump, to survive and must check their blood sugar levels regularly throughout the day. The disease can appear at any age, but it is usually diagnosed in children and young adults. Further, an individual is at higher risk for type 1 diabetes if they have a parent, brother, or sister with the disease, although many patients with type 1 diabetes do not have a family history.

Teplizumab-mzwv binds to certain immune system cells and delays progression to stage 3 type 1 diabetes, and it may deactivate the immune cells that attack insulin-producing cells while increasing the proportion of cells that help moderate the immune response. It is administered by intravenous infusion once daily for 14 consecutive days.

The safety and efficacy of teplizumab-mzwv were evaluated in a randomized, double-blind placebo-controlled trial with 76 patients with stage 2 type 1 diabetes. In the trial, patients randomly received teplizumab-mzwv 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. Additionally, the trial results showed that over a median follow-up of 51 months, 45% of the 44 patients who received teplizumab-mzwv 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 randomized to stage 3 type 1 diabetes diagnosis was 50 months for the patients who received teplizumab-mzwv and 25 months for those who received placebo. These results show teplizumab-mzwv offers a statistically significant delay in the development of stage 3 type 1 diabetes.

The most common adverse effects of teplizumab-mzwv include decreased levels of certain white blood cells, rash, and headache. Additionally, the use of teplizumab-mzwv comes with warnings and precautions, such as premedicating and monitoring for symptoms of Cytokine Release Syndrome, risk of serious infections, decreased levels of a type of white blood cell called lymphocytes, risk of hypersensitivity reactions, the need to administer all age-appropriate vaccinations prior to starting teplizumab-mzwv, and avoiding concurrent use of live, inactivated and mRNA vaccines with teplizumab-mzwv.

REFERENCE

FDA Approves First Drug That Can Delay Onset of Type 1 Diabetes. FDA. November 17, 2022. Accessed November 18, 2022. https://content.govdelivery.com/accounts/USFDA/bulletins/3389478