FDA Approves First-of-Its-Kind Drug to Delay Type 1 Diabetes

With type 1 diabetes, the body attacks itself. A person’s own immune system destroys the pancreas over time, leaving the critical organ unable to produce insulin and regulate blood sugar. Almost 1.9 million people, including about 244,000 children, live with the life-altering illness. It is a disease with no cure or means of preventing it, and the only effective treatment is lifelong monitoring and insulin injections.

But now, for the first time, the Food and Drug Administration has approved a drug to delay the inevitable. Tzield (teplizumab-mzwv) effectively slows the progression of type 1 diabetes, the agency said in a Thursday press release. The medication is administered in a series of 30-minute infusions given over 14 days. It works by glomming on to the immune cells that would otherwise target the pancreas. It might deactivate those attack cells while simultaneously boosting the number of cells that help to control immune activity, explained the FDA.

“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burden of the disease,” said John Sharretts, diabetes director in the FDA’s Center for Drug Evaluation and Research, in the news statement.

To determine the medicine’s efficacy, scientists followed 76 subjects with early stage, or pre-symptomatic, type 1 diabetes over the course of 51 months in a clinical trial. 44 of the patients were given teplizumab, while 32 received a placebo. Among the placebo patients, 72% progressed to full-blown stage 3 diabetes by the end of the trial. In comparison, only 45% of subjects given the drug ended up sick.

Without Tzield, patients got sick about 25 months into the trial. And with it, the “mid-range time” for illness onset was 50 months—a more than 2-year delay, reported the FDA.

Though it may seem small, additional years without insulin shots and blood sugar checks multiple times a day can mean a lot. Type 1 diabetes is often diagnosed in children, teens, and young adults. Managing chronic illness early in life has significant drawbacks for quality of life, according to multiple studies. For instance, diabetic children may be absent from school more often, and they are expected to manage the exhausting task of keeping tabs on their health in a way few other young people are, which takes a toll and can lead to fatigue.

Then, there’s the financial burden. Even with insurance, diabetes is an expensive disease to live with. Overall, people with diabetes in the U.S. spend an average of $9,601 every year on costs related to the illness, according to one 2018 study. Among the subset of privately insured patients with the type 1 variant, that annual cost is still $2,500 on average, according to a 2020 study.

Unfortunately, teplizumab might not help defray that cost. Provention Bio and Sanofi, the companies behind the brand name drug, said the medicine will cost $13,850 a vial, or $193,000 for a complete 14-day treatments series. Though Provention Bio touted financial assistance options in its own press release, it is unclear how or if insurance would approach coverage of Tzield. A spokesperson for the pharmaceutical manufacturers did not immediately respond to a request for more information on expected insurance coverage and out-of-pocket patient cost.

The new medication is approved for use in adults and kids at least 8 years old who currently have stage 2 type 1 diabetes. The most common side effects of Tzield were decreased white blood cell count, rash, and headache. The medication also carries an increased risk of more serious side effects like infection.

With type 1 diabetes, the body attacks itself. A person’s own immune system destroys the pancreas over time, leaving the critical organ unable to produce insulin and regulate blood sugar. Almost 1.9 million people, including about 244,000 children, live with the life-altering illness. It is a disease with no cure or means of preventing it, and the only effective treatment is lifelong monitoring and insulin injections.

But now, for the first time, the Food and Drug Administration has approved a drug to delay the inevitable. Tzield (teplizumab-mzwv) effectively slows the progression of type 1 diabetes, the agency said in a Thursday press release. The medication is administered in a series of 30-minute infusions given over 14 days. It works by glomming on to the immune cells that would otherwise target the pancreas. It might deactivate those attack cells while simultaneously boosting the number of cells that help to control immune activity, explained the FDA.

“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burden of the disease,” said John Sharretts, diabetes director in the FDA’s Center for Drug Evaluation and Research, in the news statement.

To determine the medicine’s efficacy, scientists followed 76 subjects with early stage, or pre-symptomatic, type 1 diabetes over the course of 51 months in a clinical trial. 44 of the patients were given teplizumab, while 32 received a placebo. Among the placebo patients, 72% progressed to full-blown stage 3 diabetes by the end of the trial. In comparison, only 45% of subjects given the drug ended up sick.

Without Tzield, patients got sick about 25 months into the trial. And with it, the “mid-range time” for illness onset was 50 months—a more than 2-year delay, reported the FDA.

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Though it may seem small, additional years without insulin shots and blood sugar checks multiple times a day can mean a lot. Type 1 diabetes is often diagnosed in children, teens, and young adults. Managing chronic illness early in life has significant drawbacks for quality of life, according to multiple studies. For instance, diabetic children may be absent from school more often, and they are expected to manage the exhausting task of keeping tabs on their health in a way few other young people are, which takes a toll and can lead to fatigue.

Then, there’s the financial burden. Even with insurance, diabetes is an expensive disease to live with. Overall, people with diabetes in the U.S. spend an average of $9,601 every year on costs related to the illness, according to one 2018 study. Among the subset of privately insured patients with the type 1 variant, that annual cost is still $2,500 on average, according to a 2020 study.

Unfortunately, teplizumab might not help defray that cost. Provention Bio and Sanofi, the companies behind the brand name drug, said the medicine will cost $13,850 a vial, or $193,000 for a complete 14-day treatments series. Though Provention Bio touted financial assistance options in its own press release, it is unclear how or if insurance would approach coverage of Tzield. A spokesperson for the pharmaceutical manufacturers did not immediately respond to a request for more information on expected insurance coverage and out-of-pocket patient cost.

The new medication is approved for use in adults and kids at least 8 years old who currently have stage 2 type 1 diabetes. The most common side effects of Tzield were decreased white blood cell count, rash, and headache. The medication also carries an increased risk of more serious side effects like infection.