First FDA-Approved Drug for Delaying Type 1 Diabetes Onset

April 17, 2023

Type 1 Diabetes (T1D) is a life-threatening condition that occurs when the immune system’s T cells destroy insulin-producing beta cells in the pancreas. To survive, people with T1D must take insulin for the rest of their lives. Insulin is used to control blood sugar levels and reduce the risk of severe complications.

Stages of Type 1 Diabetes

T1D progresses in three distinct stages leading up to the clinical diagnosis of disease.

Illustration of a pancreas on the left with a circle showing zooming in on the cells. Three circles, one for each stage, on the right. Stage 1 shows the beta cells with CD3 protein and T cells. Stage 2 shows this same but with fewer beta cells. Stage 3 shows fewer beta cells and no CD3 protein or T cells.

Stage 1: T cells begin to attack insulin-producing beta cells in the pancreas.
Stage 2: Some beta cells are destroyed, leading to abnormal blood sugar. T cells continue to attack remaining beta cells.
Stage 3: Nearly all beta cells are destroyed, leading to T1D symptoms.

Credit: NIAID

Journey to T1D Treatment

Since the 1970s discovery that the immune system causes T1D, scientists have worked to develop medications that prevent the immune attack.

  • 1970s

    NIH-funded and private sector scientists discovered the antibody OKT3 binds the CD3 protein on T cells.

  • 1980s

    NIH-funded investigators showed that mice treated with an OKT3-like antibody prevented T1D. However, OKT3 caused unwanted side effects in humans.

  • 1990s

    Researchers discovered how OKT3 caused unwanted side effects and designed a safer antibody.

  • 2000s

    NIH-funded clinical trials evaluated the modified, safer antibody, which was named teplizumab.

  • 2010s

    NIH-funded and industry clinical trials evaluated the safety and efficacy of teplizumab in children in Stage 2 T1D.

  • 2020s

    FDA approved teplizumab in November 2022 to delay progression of T1D from Stage 2 to Stage 3.

Decades of NIH Investment

Decades of NIH investment in basic research and clinical trials through the NIAID Immune Tolerance Network and the NIDDK T1D TrialNet, in addition to industry partnerships, led to the approval of teplizumab

How Teplizumab Works

Illustration of pancreatic cells in three stages showing how Teplizumab binds to CD3 on T cells and reduces their ability to destroy Beta cells.

Stage 1: T cells begin to attack insulin-producing beta cells in the pancreas.
Stage 2: Some beta cells are destroyed, leading to abnormal blood sugar. Teplizumab now binds to CD3 on T cells, slowing their attack.
Delayed onset: Teplizumab slows T cell destruction of beta cells, delaying onset of Stage 3 T1D.
Stage 3: Nearly all beta cells are destroyed, leading to T1D symptoms.

Credit: NIAID

First FDA-Approved Drug for Delaying T1D Onset 

Teplizumab is the first FDA-approved drug for delaying T1D onset in at-risk patients. This delay reduces the potential for severe long-term complications, thereby improving quality of life of these patients.

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