Teplizumab was given the FDA’s breakthrough therapy designation in 2019, based on initial study results showing that the drug prolonged insulin production in people with recently diagnosed type 1 diabetes and significantly delayed a diagnosis in people at high risk for type 1 diabetes. The drug works by blocking part of the immune system, stopping its attack on the insulin-producing beta cells of the pancreas. In a long follow-up analysis published last year, teplizumab was found to have a lasting effect in delaying the diagnosis of type 1 diabetes in people at high risk for the condition — based on having a close relative with type 1 diabetes and testing positive for at least two type 1 diabetes autoantibodies, proteins involved in the immune system’s attack on the pancreas. Those results prompted the FDA to give the drug priority review in its approval process.
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Tzield approved to delay type 1 diabetes
Now, Tzield is approved for adults and children ages 8 and older with what’s known as stage 2 type 1 diabetes — meaning they have at least two diabetes-related autoantibodies, and their blood glucose levels have started to become abnormal. In stage 2, people with type 1 diabetes still have a substantial amount of insulin production in their pancreas, and they often have no symptoms. Stage 3 is when people with diabetes have significant loss of pancreatic beta cells, leading to a sharp reduction in insulin production and symptoms like fatigue, unexplained weight loss, thirst, and frequent urination.
For the latest analysis that led to the approval of Tzield, 76 people with stage 2 type 1 diabetes were randomly assigned to receive either Tzield or a placebo (inactive) intravenous (IV) infusion once a day for 14 days in a row. The main outcome the researchers were interested in was the length of time from the start of the study to the development of stage 3 type 1 diabetes. During a follow-up period that lasted a median of 51 months, 45% of participants who received Tzield developed stage 3 type 1 diabetes, compared with 72% of the placebo group. The median duration from start of the study to a diagnosis of stage 3 type 1 diabetes was 50 months for participants who received Tzield, and 25 months for participants who received the placebo.
“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, in the agency’s announcement. “The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease.”
In a separate press release from Provention Bio, the developer of Tzield, the company announced the launch of a patient support program “with a staff of dedicated personnel available to answer questions and help navigate coverage, reimbursement and access for patients that are prescribed Tzield.” The company noted that it offers financial assistance for eligible patients to help with out-of-pocket costs associated with Tzield.
“It cannot be emphasized enough how precious a delay in the onset of stage 3 [type 1 diabetes] can be from a patient and family perspective,” said Ashleigh Palmer, cofounder and CEO of Provention Bio, in the press release — adding that this means “more time to live without and, when necessary, prepare for the burdens, complications and risks associated with stage 3 disease.”
As noted in an article on the approval of Tzield at MedPage Today, Provention Bio has announced that the wholesale price of each vial of the drug will be $13,300, making the total treatment cost for the drug $193,900 before any insurance coverage.