A biologic therapy that delays the onset of type 1 diabetes received approval from the US Food and Drug Administration on Thursday.
It is the first therapy approved for prevention of type 1 diabetes.
The monoclonal antibody teplizumab, which will be marketed under the brand name Tzield, from ProventionBio and Sanofi is given through intravenous infusion. The therapy will carry a wholesale cost of roughly $194,000 for a full course of treatment, the drug’s manufacturer said in an investor call on Friday, although this is not expected to be the price consumers would pay.
It is thought to work by turning down the body’s misdirected attack on its own insulin-producing cells. The idea is that protecting these cells buys people more time before they become dependent on insulin to manage their condition.
In clinical trials, Tzield delayed progression to full-blown diabetes by a little over two years. But the benefits have lasted much longer in some of the study participants.
One of them, Mikayla Olsten, was screened for diabetes after her 9-year old sister, Mia, suddenly developed a life-threatening episode of diabetic ketoacidosis and was diagnosed with diabetes. There was no history of diabetes in the family, and Mikayla wasn’t sick, but she had four of the five types of autoantibodies that doctors look for to assess a person’s risk.
“They told us when somebody has that many markers, it’s not if they’re going to develop diabetes, it’s when,” said her mom, Tracy.S
Mikayla was 15 when she joined the study and received teplizumab. She’s now 21 and a senior in college. She gets an annual battery of tests to check her pancreas and blood markers, and Tracy Olsten says her condition hasn’t progressed in six years.
According to a scientific statement from JDRF, the Endocrine Society and the American Diabetes Association, when a person has markers for autoimmune disease and episodes of uncontrolled blood sugar, the five-year risk for progression to insulin-dependent symptomatic disease is 75%. The lifetime risk of developing insulin-dependent diabetes is nearly 100%.
So far, Mikayla seems to be beating those odds.
Tracy said that for Mia, who is dependent on insulin, managing her diabetes is a constant chore.
“She has a tremendous amount of juggling that her peers don’t have to do. She has to plan ahead when she has a basketball game or practice on making sure she carbs up and decreases her insulin levels,” Tracy said. “She cannot go a minute or a day without thinking about it nonstop, and to be able to give Mikayla the opportunity where she doesn’t have to think about it 24/7 is amazing.”