November 18, 2022
Yesterday afternoon the FDA has approved the Biologics License Application for Teplizumab, an immune-modulating drug that delays the onset of type 1 diabetes (T1D). Teplizumab, which will go to market by the brand name Tzield, is the first ever disease-modifying therapy for T1D approved by the FDA.
The road to teplizumab has not been easy. In 2021, an FDA advisory committee recommended the approval of teplizumab to the FDA after a closely contested decision. At that time, the committee endorsed reservations about the decision given their uncertainties about efficacy. The FDA allowed Provention Bio to resubmit in early 2022 and, now, as of yesterday, the FDA has given the green light.
The main benefit of Tzield therapy is that if started after initial diagnosis it delays full onset. In the FDA official press release (link below), John Sharretts, M.D., director of the Divsion of Diabetes, Lipid Disorders, and Obesity at the FDA, describes Tzield this way: "The drug's potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of the disease."
Simply put, a newly diagnosed person with T1D may not be fully dependent on external insulin for up to three years, compared to 6 months for most people today. During this time, Tzield slows the auto-immune progression allowing the natural ability to make insulin to continue longer and moderately reducing the risk of long term complications down the road.
That said, Tzield delays but does not stop or prevent the progression of the disease. Eventually, patients will require insulin injections for life. It is distinctly not a functional cure.
Tzield has no impact on a person who is already a fully-established T1D.
WHAT IS THE PATIENT EXPERIENCE?
Tzield will be available to adults and pediatric patients 8 years and older. It is administered to patients by an intravenous infusion, lasting a minimum of 30 minutes, one time per day over a period of 14 days.
WHAT DOES THIS MEAN FOR CURE PROGRESS?
Teplizumab is a man-made monoclonal antibody that binds to the CD3 receptor on T cells to reduce the autoimmune attack. While teplizumab is not a cure and has not been shown to prevent T1D, it is the first drug brought to market that can change the biological course of the disease.
For many, teplizumab represents a renewed hope for a long-promised cure in the future. They hope that this breakthrough will expand, develop and evolve to provide immune modification for the long run. And, they might envision, if combined with a source of cell supply, perhaps it could help unlock a future pathway to a practical cure. But these ideas are hopeful conjecture and have distinctly not been stated as objectives by Tzield's parent company, Provention Bio.
For others with longstanding T1D, teplizumab is much less exciting since it has no impact on those currently living with the disease.
ROUTE TO MARKET AND PATIENT ACCESS
For Provention Bio, there is still much to do in the road ahead, including continued work with the FDA, scale-up manufacturing, distribution and physician sales/training. A co-promotion partnership with Sanofi for the U.S. launch of Tzield will give the new drug access to Sanofi's infrastructure and expertise.
Patient's interested can discuss the therapy with their medical professional. Provention Bio also has a program to help manage costs for those in need.