Teplizumab FDA approval for diabetes: TZIELD delays onset of type 1 – USA TODAY

When Mikayla Olsten was a teenager, she watched her 10-year-old sister nearly die from diabetic ketoacidosis. Nobody knew her sister had Type 1 diabetes, and as a result, the entire family got screened.
That’s when Olsten found out she had pre-diabetic markers. At 15, doctors said she had about six months before fully developing the lifelong disease.
“I was really, really scared,” said Olsten, who turns 21 next week and is a student living in Idaho. “I practically almost saw my sister die. … And I’m like, ‘If I have this, I could die.’”
But there was hope. She entered a clinical trial in Gainesville, Florida, where researchers were studying a treatment that could delay the onset of Type 1 diabetes. Six years later, Olsten isn’t dependent on insulin.
The Food and Drug Administration on Thursday approved the monoclonal antibody teplizumab, which will be sold under the brand name TZIELD, possibly saving patients like Olsten years of having to manage a costly and burdensome chronic disease. 
“It is game-changing,” said Dr. Mary Pat Gallagher, director of the Pediatric Diabetes Center at Hassenfeld Children’s Hospital at NYU Langone. “There are almost 2 million people in the United States who have Type 1 diabetes now, and it’s increasing in frequency.”
The kicker: The cost is $13,850 a vial for a total of $193,000 over the 14-day treatment, a notable price given the recent attention over the escalating cost of insulin. A study published in October found more than 1.3 million U.S. adults skipped doses, delayed buying or otherwise rationed the lifesaving medication insulin because of its cost.
A spokesperson for ProventionBio and Sanofi, the pharmaceutical companies behind the new treatment, did not immediately respond when asked about the price and insurance coverage.
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Type 1 diabetes is an autoimmune disorder that causes the destruction of cells that secrete insulin from the pancreas, according to the British Diabetic Association, or Diabetes UK. It is different from Type 2 diabetes, which is caused when the body makes less insulin or becomes resistant to insulin, often because of excess body fat.
The FDA approved the treatment as a 14-day, 30-minute infusion for adult and children 8 years and older with Stage 2 Type 1 diabetes. Teplizumab will be available nationwide by the end of this year, according to Sanofi 
In the three stages of Type 1 diabetes, Stage 2 is one step before clinical diagnosis. In clinical trials, the newly approved treatment delayed Stage 3 disease onset for about two years compared with the placebo.
Stage 2 is marked by having diabetes-related antibodies and abnormal levels of blood sugar. But patients don’t typically experience symptoms until they’re diagnosed with Stage 3.
Health experts say delaying the final stages of Type 1 diabetes could save patients years of taking insulin, counting foods and monitoring blood sugar.
Olsten said she couldn’t have imagined doing all that while also finishing high school and navigating college. 
“It was already hard enough,” she said. “If I had to go to doctors constantly or go prick my finger or insert insulin, I don’t think I would feel as normal as I would have when I did go through high school.” 
The treatment could also save patients thousands of dollars, experts say. The American Diabetes Association estimates people diagnosed with diabetes spend an average of $16,752 on medical expenses a year, with more than $9,600 directly attributed to diabetes. 
“I don’t think people actually understand how much people’s lives get changed with diabetes,” said Dr. Kathleen Bethin, clinical professor pediatrics at the State University of New York at Buffalo.
One of the side effects of TZIELD is a decrease in a white blood cell count, which health experts say could increase the risk of developing severe disease from opportunistic infections. But the trial showed cell counts began rising again seven days after treatment and returned to pre-treatment counts a few months later.
Experts say it may be a small price to pay for reducing the risk of developing serious complications from diabetes like kidney disease or ketoacidosis – like Olsten’s sister. Diabetic ketoacidosis is an overload of ketones present in the blood and can lead to a diabetic coma or even death. 
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Although patients who take the treatment will likely develop Type 1 diabetes at some point in their lives, health experts hope Thursday’s approval marks the start of learning how to prevent the disease instead of just managing it.
“This is a historic moment for all people affected by Type 1 diabetes,” said Dr. Robert Gabbay, chief scientific and medical officer of the American Diabetes Association. “The immeasurable benefits of improved quality of life will be felt not only by those diagnosed with (Type 1 diabetes), but also by their families.” 
Follow Adrianna Rodriguez on Twitter: @AdriannaUSAT. 
Health and patient safety coverage at USA TODAY is made possible in part by a grant from the Masimo Foundation for Ethics, Innovation and Competition in Healthcare. The Masimo Foundation does not provide editorial input.
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