FDA approves first treatment to delay onset of type 1 diabetes


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 the prevention of type 1 diabetes.

The monoclonal antibody teplizumab, which will be marketed under the brand name Tzield from ProventionBio and Sanofi, is given by intravenous infusion.

It is thought to work by reversing a misdirected attack on the body’s own insulin-producing cells. The idea is that protecting these cells gives people more time to manage their condition than if they were dependent on insulin.

In clinical trials, Tzield delayed the transition to full-blown diabetes by a little over two years. But in some study participants, the benefits lasted longer.

One of them, Mikayla Olsten, was tested 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 family history of diabetes, and Mikayla was not sick, but she had four of the five types of autoantibodies that doctors look for to assess a person’s risk.

“They told us that if someone has that many markers, it’s not that they’re going to develop diabetes, it’s when,” said her mother, Tracy.

Mikayla was 15 years old when she entered the study and received teplizumab. He is now 21 years old and in college. He receives an annual battery of tests to check his pancreas and blood markers, and Tracy Olsten says his condition hasn’t improved in six years.

According to a scientific statement from the Juvenile Diabetes Research Foundation, the Endocrine Society and the American Diabetes Association, when a person has markers for autoimmune disease and episodes of uncontrolled blood sugar, the risk of developing symptomatic insulin-dependent disease is five years. 75%. The lifetime risk of developing insulin-dependent diabetes is approximately 100%.

So far, Mikayla is beating the odds.

Managing diabetes is a constant task for Mia, who is insulin dependent, Tracy said.

“He has a great juggling act that his peers don’t have to do. She has to plan ahead when she has a basketball game or when she’s working out to make sure she’s increasing her carbs and lowering her insulin,” Tracy said. “She can’t go a minute or a day without thinking about it non-stop and giving Mikayla an opportunity where she doesn’t have to think about it 24/7 wonderful to know.”

Aaron Kowalski, CEO of the Juvenile Diabetes Research Foundation, says the main challenge in prescribing Tzield will be finding people who need it. The drug is approved for people who have no symptoms of the disease and do not know they are on the way to contracting the disease.

“Screening becomes a really big problem because, as far as we know, about 85% of type 1 diagnoses today are in families with no known family history,” Kowalski said. “Our goal is to do general population screening” with blood tests to look for markers of the disease.

Tzield is approved for use in people 8 and older with stage 2 type 1 diabetes. At this stage, doctors can measure antibodies in the person’s blood that attack the insulin-producing beta cells, and they have abnormal blood sugar levels, but their body is still able to produce insulin.

“The way not only the industry, but also our medical system, manages autoimmune diseases, and type 1 diabetes in particular, is truly suboptimal in today’s day and age,” said Ashleigh Palmer, co-founder and CEO of ProventionBio. “What we do is we wait until the symptoms of the disease come to the doctors, and then the doctors treat the patient’s symptoms chronically for life. The problem is that when symptoms first appear in type 1 diabetes, it’s already too late.”

Treatment comes in a single course of 14-day infusions lasting 30 to 60 minutes each.

The most common side effects reported in trial participants were low white blood cells and lymph cells, rash, and headache.

With type 1 diabetes, a person’s immune system attacks cells in the pancreas called beta cells that produce insulin, a hormone that helps blood sugar enter cells where it is used for energy. An attack can occur years before any signs of diabetes appear. Without insulin, blood sugar can build up in the bloodstream and break down the body’s own fat and muscle.

Palmer says Tzield stops the disease before symptoms appear by stopping the autoimmune disease process and the underlying destruction of beta cells. The treatment basically reboots the immune system while preserving beta cell function.

“We really don’t have any preventative measures for type 1 diabetes to date, and yet [the National Institutes of Health] “A program called TrialNet, hundreds of millions of dollars in funding over the last 20 years, has tested a lot of different things, including this, and some of that came out of this,” he said. Medical Officer of the American Diabetes Association. “Finally, there’s something that delays the onset of type 1 diabetes, and that’s very exciting.”

Unlike type 2 diabetes, which can be prevented with lifestyle changes such as losing weight and exercising, type 1 is a genetic disease with no prevention to date.

“For some reason, we don’t screen for type 1 diabetes, even though there are biomarkers available to indicate that the autoimmune disease process is already underway,” Palmer said. He added that hopes will catalyze the medical system to begin population-based screening during regular childhood visits to prevent the disease and delay its onset.

With Tzield, doctors will test individual family members of people with type 1 diabetes to see if they have these specific antibodies. If antibody levels are high and a person appears to be about to develop diabetes, treatment slows the process.

“If someone has type 1, a common question is, ‘Well, what about my child?’ Will they develop type 1?’ It’s only a 5% risk, so they won’t happen often, but if you can find the ones to treat them, it can make a big difference,” Gabbay said.

A delayed diagnosis of type 1 diabetes can have a significant impact.

“Obviously, if you’re diagnosed with type 1 diabetes, your quality of life is significantly impacted, negatively impacted. It’s a disease that never goes away,” Palmer said.

Type 1 diabetics must monitor their blood glucose levels around the clock, which affects how they exercise and eat. High blood sugar can lead to diabetic ketoacidosis, in which the body begins to break down fat for fuel and can lead to a build-up of acids called ketones in the blood. This condition can lead to hospitalization, coma, or death.

As of 2019, approximately 1.9 million people in the United States, including 244,000 children and adolescents, have type 1 diabetes, according to the American Diabetes Association. It affects 8% of all people with type 1 diabetes.

“The incidence of type 1 is mostly in children and adolescents, and when you’re in the throes of adolescence, you just want to forget you have it,” said Olivier Bogillot, Sanofi’s head of US generics. “So when you can with a treatment to delay the onset of the disease, you can change the impact on the families and the quality of life for these children.”

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