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The U.S. Food and Drug Administration (FDA)’s regulatory review is now under way for teplizumab to delay clinical type 1 diabetes (T1D). Today, an FDA Advisory Committee weighed the evidence, and the vote was positive: The advisory committee recommended that the FDA approve teplizumab for at-risk individuals, with 10 members voting yes and 7 members voting no. (At-risk means that they exhibited abnormal glucose levels and at least 2 T1D-related autoantibodies, but who have not been diagnosed with the disease.)

The advisory committee heard from Aaron Kowalski, Ph.D., CEO of Breakthrough T1D, who stressed the unmet need for disease-modifying therapies—which can slow, halt, or reverse the course of the disease—for T1D. Breakthrough T1D also submitted a letter to the committee in advance of the meeting.

“A delay in onset of clinical type 1 diabetes will have long-term benefits for blood-sugar control, and the reduction in acute and long-term complications would have a tremendous impact on the daily lives of people with type 1 diabetes, their families, and our overall health system,” said Dr. Kowalski. “Beyond the important and significant clinical benefits, any person with type 1 diabetes and their family can convey the profoundly positive impact a delay in onset would have on their daily lives, and how they feel, function, and survive. It would free them from the constant burden and stress of glucose monitoring and insulin administration. It would free them from the worry and fear of short- and long-term complications while giving them the opportunity to learn more about disease management. And it would allow them to live life like those of us without type 1 diabetes are able to do. That is clinically meaningful.”

If determined by FDA to be safe and effective, teplizumab can aid in addressing the stark unmet need for disease-modifying therapies and provide people at-risk for T1D and their families at least two more years without the burden and complications this disease brings. Disease-modifying therapies put us on the critical pathway to preventing and ultimately curing T1D.

Breakthrough T1D thanks the committee for their thorough and considerate review of the evidence and future impact of teplizumab. If approved, teplizumab will be the first disease-modifying drug for individuals at-risk for developing T1D. The target action date by the FDA is July 2, 2021, however the sponsor, Provention Bio, has stated it will likely be delayed.

Breakthrough T1D had a hand in the development of teplizumab from nearly the beginning:

  • Breakthrough T1D was one of the first to support preclinical and early clinical trials to see if teplizumab worked for people with T1D.
  • We gave a Career Development Award to Kevan Herold, M.D., who had just started his faculty-level career at The University of Chicago, in 1988-1990. He showed, in an early animal study, that he could prevent autoimmune diabetes with an anti-CD3 antibody (which, later, became a humanized version, teplizumab). He has gone on to receive more than 15 grants from Breakthrough T1D, and was the lead on the clinical trial that demonstrated that teplizumab could delay the onset of clinical T1D for nearly three years in people almost certain to develop the disease. This was the first ever study in humans to show a delay in the onset of T1D.
  • We funded clinical trials in recent-onset disease, which were published in 2002, 2005, and 2013, and a phase III trial run by MacroGenics, who received a Breakthrough T1D Industry Discovery and Development Program grant from 2006-2011.
  • In 2012-2019, we funded, with the National Institutes of Health (NIH), the Immune Tolerance Network—an international clinical research consortium—which did a clinical trial of teplizumab and identified who is likely to respond to the drug. We also funded, with the NIH (using funds from the Special Diabetes Program) and the American Diabetes Association, TrialNet—the largest clinical network for T1D—who ran the clinical trial in at-risk individuals, led by Dr. Kevan Herold.
  • In 2017, the Breakthrough T1D T1D Fund invested in Provention Bio to facilitate the acquisition and clinical development of T1D relevant assets. The T1D Fund no longer has a financial interest in the company.

If successful, and the FDA review results in approval, we will be moving ever closer to a world without this disease.