Provention Bio’s patient support program helps commercially insured patients and patients without insurance coverage who qualify to pay $0 out of pocket for Tzield. Find out more about their patient support program at Tzield.com.
The Food and Drug Administration (FDA) on Thursday approved Tzield (teplizumab-mzwv), Provention Bio’s immune therapy indicated to delay the onset of type 1 diabetes (T1D).
Teplizumab, an anti-CD3 monoclonal antibody, is the first approved therapy shown to delay the progression to stage 3 T1D for some people at high risk of developing the condition. The therapy is approved to treat adults and children 8 years and older who live with stage 2 T1D.
Teplizumab is administered by intravenous infusion once daily for 14 consecutive days.
“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease,” said Dr. John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research.
Teplizumab binds to certain immune system cells and delays the progression of T1D. The therapy may deactivate the immune cells that attack insulin-producing beta cells, while increasing the proportion of cells that help moderate the immune response, essentially stabilizing beta cell function.
Autoantibodies are antibodies made by your immune system that attack your own tissue and are often associated with autoimmune reactions. T1D-related autoantibodies can show up in blood tests months or years before the onset of the physical symptoms that typically lead to a diagnosis.
Clinical trials of teplizumab focused on participants who were already experiencing a decline in c-peptide levels, meaning that insulin production and beta cell function were already being impacted.
The median time from joining the study to the onset of stage 3 T1D was 50 months for the group that received teplizumab, versus 25 months for the group that did not receive the therapy.
The list price of a 14-day infusion course of Tzield costs $193,900, or $13,850 per vial, according to Provention Bio. The manufacturer’s patient support program, Provention Bio COMPASS, aims to help bring the cost to $0 for commercially insured patients and patients without insurance coverage who qualify.
More time with preserved hormone function and stable blood sugar levels means less strain on the body caused by blood sugar fluctuations. Delaying the onset of T1D also means less time spent having to manage the condition and mitigating its risks and complications.
“This is a historic occasion for the T1D community and a paradigm-shifting breakthrough for individuals aged 8 years and older with Stage 2 T1D who now have a therapy approved by the FDA to delay the onset of Stage 3 disease,” Provention Bio co-founder and CEO Ashleigh Palmer said.
There are other long-term benefits to slowing the loss of beta cell function. By actively monitoring autoantibody levels and other markers of T1D in individuals who receive this preventative treatment, it’s possible to minimize the risk of–or even prevent—diabetic ketoacidosis (DKA).
DKA is a life-threatening complication of diabetes and persistently high blood sugar that often leads to hospitalization.
Many newly diagnosed individuals are in DKA at the time of their T1D diagnosis, including up to 40% of newly diagnosed children. (Children make up about half of all new T1D cases.)
Avoiding DKA is vital—and DKA at the time of diagnosis is associated with worse long-term diabetes-related outcomes.
Many years of research were behind the approval of this breakthrough treatment. In fact, Dr. Kevan Herold, chair of TrialNet, the clinical network dedicated to T1D that ran the teplizumab trial, first demonstrated that an anti-CD3 antibody could prevent T1D more than 20 years ago.
“The recent decision represents a turning point in the field,” Herold said in a statement Thursday. “First, it identifies a way in which an immune therapy to stop the disease process might be combined with cell replacements in those with type 1 diabetes. It also suggests that it is time to more broadly screen to identify those at risk for type 1 diabetes since now there is a therapy that can change its course.”
JDRF is a leader in funding T1D research and was an early supporter of the work that lead to Tzield. JDRF celebrated the approval and reflected on what it means for families.
“A delay in the onset of type 1 diabetes will have a tremendous impact on the daily lives of people at risk for diabetes, their families, and the overall health system,” said JDRF CEO Aaron Kowalski. “It would free them from the constant burden and stress of blood-sugar 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. That is clinically meaningful.”
Both participants and their families spoke out about the benefits they’ve gained from teplizumab treatment. Tracy Olsten enrolled her daughter, Mikayla, who was positive for autoantibodies.
“We know the probability is high that she will develop T1D in her life. But a little bit more time to live her teenage, young adult, and adult years without injections and finger pokes,” Olsten told JDRF.
Last month, global pharmaceutical company and insulin manufacturer Sanofi secured its spot for the first negotiations for the exclusive global rights to research, develop and commercialize teplizumab in a $20 million agreement with Provention Bio.
Sanofi will hold the right to negotiate through June, with a provision for a possible extension through 2023. Industry experts suspect Sanofi may look to extend negotiations to coincide with the results of the phase 3 PROTECT trial.
Positive results from PROTECT could lead to teplizumab getting approved for wider use.
The ongoing PROTECT study, slated to be completed in May, looks at whether teplizumab preserves beta cell function in children recently diagnosed with T1D.
The trial will include 300 participants between the ages of 8 and 17 who have been diagnosed with T1D within the last six weeks at the time of their enrollment. Participants will receive two 12-day courses of teplizumab six months apart.
The FDA declined Provention Bio’s application for teplizumab in June 2021, requesting more information. This followed an FDA advisory committee’s narrow vote to recommend teplizumab’s approval.
Teplizumab falls under a newer class of medications called biologics, which are more complex in how they work inside the body compared to traditional, small-molecule drugs. It’s common for the FDA to request more documentation on safety, efficacy and how a treatment works.
Regulators accepted teplizumab’s safety and efficacy data but requested blood sample data over time demonstrating that the human body interacted with the latest version of the therapy—the version under review—comparably to the version of teplizumab used in trials.
The FDA granted Breakthrough Therapy Designation to teplizumab in 2019 after it became the first drug ever to demonstrate the delay of T1D onset successfully.
This designation is given to treatments for serious or life-threatening conditions where clinical trials have shown significant advantages over existing therapies, though it does not guarantee FDA approval. In this program, a drug manufacturer can get guidance and support to speed up a therapy’s development, with the goal of making it available to the public faster.
Editor’s note: This article has been updated.
