FDA approves ‘game changing’ treatment that delays Type 1 diabetes

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. She was 15, and 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.

“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 companies behind the new treatment, did not immediately respond when asked about the price and insurance coverage.

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 adults and children 8 years and older with Stage 2 Type 1 diabetes.

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. The treatment also could save patients thousands of dollars, experts say.

One of the side effects of TZIELD is a decrease in a white blood cell count, which experts say could increase the risk of developing severe disease from opportunistic infections. But the trial showed cell counts returned to pretreatment counts a few months later.

“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.”

Dr. Robert Gabbay Chief scientific and medical officer of the American Diabetes Association