FDA Breakthrough Therapy Designation for Teplizumab—Based on the First Study to Delay the Onset of T1D for 2+ Years

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The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to teplizumab, an anti-CD3 monoclonal antibody, for the prevention or delay of type 1 diabetes (T1D) in individuals at-risk of developing the disease. CD3 is a blood marker that helps to activate the immune cells—called T cells—which are thought to be responsible for the disease.

Kevan Herold, M.D., of Yale University and a longtime JDRF grantee, presented the results of the clinical trial at the American Diabetes Association’s Scientific Sessions in June. For the first time ever, an immune therapy treatment was able to significantly delay—for over two years—the onset of T1D in participants with a high risk of developing the disease. The FDA’s decision to provide Breakthrough Designation recognizes the importance of these results to the T1D community, and those at risk of developing it, and is intended to lead to an interactive and efficient drug development process. JDRF will continue to fight for the development of teplizumab and other treatments that show the potential to make a significant difference to people with—and at risk for—T1D.

JDRF funded one of the first clinical trials of this drug, results of which were integral to the teplizumab prevention trial. This study was conducted by TrialNet, also funded by JDRF and by the NIH’s Special Diabetes Program for which JDRF is the leading advocate.

Teplizumab (also called PRV-301) is now being tested in a phase III clinical trial in people recently diagnosed with T1D, sponsored by Provention Bio, a company with an investment from the JDRF T1D Fund.

If successful, this could become an approved therapy that modifies the disease course of T1D, instead of only treating the symptoms.