Teplizumab approved for use in people at risk of type 1 diabetes by the US Food and Drug Administration

This is the first therapeutic on the market
that may help delay the onset of clinical
type 1 diabetes

November 17, 2022

Today, the U.S. Food and Drug Administration (FDA) weighed the evidence for teplizumab to delay type 1 diabetes (T1D) in at-risk individuals, and the T1D community got a big win: Approved! Teplizumab (Provention Bio brand name Tzield) is the first disease-modifying therapy for individuals at-risk for developing T1D. (At-risk means that they exhibited 2+ T1D-related autoantibodies—antibodies against one’s own self—and their blood glucose is starting to be abnormal, but they have not yet become insulin dependent.)

For the first time in history, there is an approved therapy that addresses the autoimmunity behind T1D—not just the symptoms that it causes.

Teplizumab can start to address 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. Teplizumab, and multiple potential disease-modifying therapies that JDRF has invested research into, put us on the critical pathway to finding cures and, one day, preventing T1D entirely.

JDRF and all its affiliates thank the FDA for their thorough and thoughtful review of the teplizumab evidence and data, showing that the benefits of teplizumab outweigh the risks to delay clinical T1D in at-risk individuals.

A day decades in the making

The approval of teplizumab would simply not have been possible without decades of JDRF work, from funding discovery research to clinical development to work with regulators. JDRF had a hand in the development of teplizumab from the very beginning.

The JDRF T1D fund made a strategic investment in Provention Bio in 2017 that brought the company into T1D for the first time. That investment has helped catalyze hundreds of millions of dollars toward clinical development, regulatory work, and launch preparation that made this moment possible.

Teplizumab is now the first ever disease-modifying therapy for T1D. In clinical trials led by TrialNet and supported by JDRF and other funders, teplizumab was able to significantly delay—for over two years—the onset of T1D in participants with a high risk of developing the disease, the first time a study in humans demonstrated a delay in the onset of T1D.

In long-term follow up of trial participants,  the effect of teplizumab was even more striking: 50% of those treated with teplizumab remain diabetes-free, compared to only 22% of those taking placebo, and the delay in diabetes onset was close to 3 years (35 months).

Additionally, the participants, in both the teplizumab and placebo groups, had had a progressive decline in the biomarker that measure’s the body’s ability to produce insulin—C-peptide—preceding the trial. But the study team found that production of C-peptide went up following treatment with teplizumab, particularly in the first 6 months after treatment. Teplizumab could potentially reverse the downward trajectory of C-peptide loss that was there before the trial.

A key tenet of JDRF’s research funding strategy is that there are multiple pathways to cures for T1D, one of which includes screening for risk and use of therapeutics to delay or even prevent the onset of the disease developing – to give more time to the individual to adapt before requiring external insulin.

On Thursday, October 6, 2022, Provention Bio announced that it was partnering with pharmaceutical manufacturer Sanofi to support the US launch of teplizumab.

This co-promotion agreement will enable Provention Bio to leverage Sanofi’s substantial expertise, capabilities and commercial resources to bring the drug to those in the US in need with high efficiency.

The importance of screening

JDRF, through research grants, T1D Fund investments, and advocacy, has accelerated multiple potential disease-modifying therapies for T1D that may help to move the field towards this goal. Screening is the only way that individuals can determine their risk and to find out if they are candidates for such therapies.

Canadians who have a first-degree family member with T1D can currently be screened via TrialNet. Research has shown that family members of people with T1D are at a 15x greater risk of developing T1D than the general population. The goal for this program aligns with JDRF’s research strategy of identifying preventative and screening measures that will stop the disease before it starts.

JDRF Canada has also launched a funding opportunity, in partnership with the Canadian Institutes of Health Research (CIHR), for a T1D Screening Research Consortium to be announced in summer 2023.

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