A key tenet of JDRF’s research funding strategy is that there are multiple pathways to cures for type 1 diabetes (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 will partner with pharmaceutical manufacturer Sanofi to support the launch of teplizumab, if it is approved for use in the general population.
What does this partnership mean?
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.
The importance of screening
Canadians who have a 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.
A biologic drug called teplizumab has shown great promise as 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 type 1 diabetes (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, and 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.
The drug, now being developed by Provention Bio, was submitted to the US Food and Drug Administration (FDA) in early 2021 – and was the first disease-modifying drug ever submitted for approval to the regulator for use in T1D. On May 27, 2021, an advisory committee to the FDA recommended that teplizumab be approved for prevention of T1D.
However, on July 2, 2022, the FDA issued a Complete Response Letter to Provention Bio,. but ultimately the drug was not approved, and the company was asked to provide more data on issues concerning manufacturing before resubmitting for approval
As of October 2022, teplizumab is again under review at the FDA. The FDA has until November 17th to finish its review of the application.
As a part of this co-promotion agreement, Sanofi will bring its extensive network of industry leaders and sales and marketing team to Provention Bio, meaning it can support increased screening and help raise awareness for teplizumab which, if approved, will be the first-ever disease modifying therapy for T1D.
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 led to the partnership with Sanofi.
This partnership demonstrates an endorsement of the potential for T1D disease-modifying therapies from one of the world’s leading pharmaceutical companies. Moreover, if teplizumab is approved by the FDA, the agreement will allow more people to learn about the drug and ideally benefit from it.
JDRF’s T1D Fund has more than 20 active cures programs in development. Sanofi’s support of teplizumab gives us confidence that large pharmaceutical companies will invest more readily in these ground-breaking programs and help drive toward cures faster.
Learn more at the press release.
JDRF Canada will continue to follow the results of the FDA application review and provide updates as to when teplizumab may be available for use in Canada.