Delaying the disease can also delay any onset of vision problems, including diabetic retinopathy and diabetic macular edema.
The FDA today announced the approval of teplizumab-mzwv (Tzield; ProventionBio/Sanofi) injection, a drug that delays the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes. The development could have a ripple effect through ophthalmology.
Diabetes can damage the eyes over time and cause vision loss, even blindness. Delaying the disease can also delay any onset of vision problems, including diabetic retinopathy and diabetic macular edema.
John Sharretts, MD, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research, noted in an FDA news release that the approval of a first-in-class therapy adds an important new treatment 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 said in the news release.
The monoclonal antibody teplizumab-mzwv drug is thought to work by turning down the body’s misdirected attack on its own insulin-producing cells. The idea is that protecting these cells buys people more time before they become dependent on insulin to manage their condition.
The FDA noted that Type 1 diabetes is a disease that occurs when the immune system attacks and destroys the cells that make insulin. People with a type 1 diabetes diagnosis 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.
Moreover, the FDA also noted that while it can appear at any age, type 1 diabetes is usually diagnosed in children and young adults. A person is at higher risk for type 1 diabetes if they have a parent, brother or sister with type 1 diabetes, although most patients with type 1 diabetes do not have a family history.
Unlike type 2 diabetes, which in some instances can be prevented with lifestyle changes like losing weight and exercising, type 1 is a genetic disease that has not had any prevention options until now.
The development ultimately could impact ophthalmology, with diabetic retinopathy the leading cause of blindness in working-age adults.
According to the news release, teplizumab-mzwv binds to certain immune system cells and delays progression to stage 3 type 1 diabetes. Teplizumab-mzwv may deactivate the immune cells that attack insulin-producing cells, while increasing the proportion of cells that help moderate the immune response. Teplizumab-mzwv is administered by intravenous infusion once daily for 14 consecutive days.
The FDA noted that teplizumab-mzwv’s safety and efficacy were evaluated in a randomized, double-blind, event-driven, 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. 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 randomization to stage 3 type 1 diabetes diagnosis was 50 months for the patients who received teplizumab-mzwv and 25 months for those who received a placebo. This represents a statistically significant delay in the development of stage 3 type 1 diabetes.
According to the news release, common side effects of teplizumab-mzwv include decreased levels of certain white blood cells, rash and headache. The use of teplizumab-mzwv comes with warnings and precautions, including 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; as well as avoiding concurrent use of live, inactivated and mRNA vaccines with teplizumab-mzwv.