On November 17, the US FDA announced the approval of teplizumab (Tzield), which is administered through IV infusion once daily for 14 consecutive days, for delaying the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes.
The fight against diabetes has taken a historic turn, with the US Food and Drug Administration (FDA) approval of teplizumab (Tzield) injection to delay the onset of type 1 diabetes in adults and pediatric patients.
Announced in a statement on November 17, the FDA’s approval indicates the anti-CD3 antibody from Provention Bio, which will be marketed through a partnership with Sanofi, for delaying the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years and older who currently have stage 2 type 1 diabetes.
“Today’s approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients,” said John Sharretts, MD, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research. “The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease.”
With approval based on data from a phase 2 trial examining the agent, which is delivered through an IV infusion once daily for 14 consecutive days, represents a turning point for the diabetes community. Data from the pivotal phase 2 trial was published in the New England Journal of Medicine in 2019. The study, which included 76 patients, with the majority being under 18 years of age, produced results indicating the median time to diagnosis in the intervention arm was 48.4 months compared to 24.4 months in placebo arm. In a median of 2 years of follow-up, type 1 diabetes was diagnosed in 43% of teplizumab arm and 72% of placebo arm (HR, 0.41 95% CI, 0.22-0.78; P=.006).
Extended follow-up data from this trial, which was published in Science Translational Medicine in March 2021, suggests the median time to clinical type 1 diabetes diagnosis was 59.6 months with teplizumab arm and 27.1 months in the placebo arm. In a median of 2.5 years of follow-up, type 1 diabetes was diagnosed in 50% of the teplizumab arm and 78% of the placebo arm (HR, 0.457; P=.01).
In May 2021, teplizumab was the subject of an FDA Endocrinologic and Metabolic Drugs Advisory Committee Meeting. In this meeting, the FDA committee concluded with a 10 to 7 vote in support of teplizumab, based on the benefits detailed in background documents and presentations, for delaying clinical type 1 diabetes.
In their release, the FDA noted the most common adverse events seen teplizumab were decreased levels of certain white blood cells, rash, and headache. The FDA statement also points out the agent is accompanied by specific warnings and precautions for some patient groups, including premedicating and monitoring for symptoms of Cytokine Release Syndrome, risk of serious infections, decreased levels of a type of white blood cell called lymphocytes, and risk of hypersensitivity reactions.
"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. It cannot be emphasized enough how precious a delay in the onset of Stage 3 T1D can be from a patient and family perspective; more time to live without and, when necessary, prepare for the burdens, complications and risks associated with Stage 3 disease" noted Ashleigh Palmer, Co-Founder and CEO of Provention Bio, in a statement from Provention Bio.
For more insight into the approval of teplizumab and what it means for providers and patients, check out this special edition episode of Diabetes Dialogue: Therapeutics, Technology, & Real-World Perspectives.