FDA News Release: Published on November, 2022

Type one diabetes mellitus (T1DM) is an autoimmune disease characterized by gradual destruction of beta cells in islets of Langerhans. Teplizumab is a humanized anti- CD3 monoclonal antibody, which have beneficial effects for T1DM patients.

The U.S. Food and Drug Administration (FDA) has approved teplizumab injection to delay 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 monoclonal antibody teplizumab, which will be marketed under the brand name Tzield, is administered by intravenous infusion once daily for 14 consecutive days. 

This 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.

TAKE HOME MESSAGE

Teplizumab is the first disease-modifying therapy in Type 1 Diabetes, a life-threatening autoimmune disease

In a clinical trial, in Stage 2 T1D patients, Teplizumab delayed the median onset of Stage 3 T1D by 25 months, or approximately 2 years, compared to placebo

Stage 3 T1D is associated with significant health risks, including diabetic ketoacidosis, which can be life threatening

Patients who progress to Stage 3 T1D eventually require insulin injections for life

Mechanism of action:

Teplizumab binds to certain immune system cells and delays progression to stage 3 type 1 diabetes. Teplizumab may deactivate the immune cells that attack insulin-producing cells, while increasing the proportion of cells that help moderate the immune response.

Safety and Efficacy:

Teplizumab’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 or a placebo once daily via intravenous infusion for 14 days.

The primary measure of efficacy was the time from randomization to the 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 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 and 25 months for those who received a placebo. This represents a statistically significant delay in the development of stage 3 type 1 diabetes. 

Side Effects:

Most common adverse reactions (>10%) were lymphopenia, rash, leukopenia, and headache.

Precautions:

The use of Teplizumab 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; as well as avoiding concurrent use of live, inactivated and mRNA vaccines with Teplizumab.