A biologic therapy that delays the onset of type 1 diabetes received approval from the U.S. Food and Drug Administration on Thursday.
It is the first therapy approved for the prevention of type 1 diabetes.
The teplizumab monoclonal antibody, which will be marketed under the Tzield brand, from ProventionBio and Sanofi is administered by intravenous infusion. The therapy will incur a wholesale cost of about $194,000 for a full treatment, the drugmaker said in an investor call on Friday, though that’s not the price consumers would pay.
It is thought to work by decreasing the body’s misdirected attack on its own insulin-producing cells. The idea is that protecting these cells buys people time before they become dependent on insulin to manage their condition.
In clinical trials, Tzield delayed progression to full-blown diabetes by just over two years. But the benefits 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 family history of diabetes and Mikayla was not sick, but she had four of the five types of autoantibodies doctors look for to assess a person’s risk.
“They told us when someone has that many markers, it’s not if they’re going to get diabetes, it’s when,” said his mother, Tracy.
Mikayla was 15 when she joined the study and received teplizumab. She is now 21 years old and in high school. She undergoes a battery of annual tests to check her pancreas and blood markers, and Tracy Olsten says her condition has not progressed in six years.
According to a scientific statement from JDRF, the Endocrine Society, and the American Diabetes Association, when a person has markers of autoimmune disease and episodes of uncontrolled blood sugar, the five-year risk of progression to symptomatic insulin-dependent disease is by 75%. The lifetime risk of developing insulin-dependent diabetes is almost 100%.
So far, Mikayla appears to be beating those odds.
Tracy said that for insulin-dependent Mia, managing her diabetes is a constant chore.
“She has a lot of juggling that her peers don’t have to do. She needs to plan ahead when she has a basketball game or practice to make sure she’s upping her carbs and lowering her insulin,” Tracy said. “She can’t 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.”
Aaron Kowalski, CEO of JDRF, 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 may not know they are about to catch it.
“Testing is becoming a very big issue, because what we know is that about 85% of type 1 diagnoses today are in families who don’t have a known family history,” Kowalski said. “Our goal is to screen the general population” with blood tests to look for markers of the disease.
Tzield is approved for use in people 8 years of age and older who have stage 2 of their type 1 diabetes. At this stage, doctors can measure antibodies that attack insulin-producing beta cells in the blood of the person, and they have abnormal blood sugar levels, but their body can still produce insulin.
“The way not only the industry, but also our medical system, deals with autoimmune diseases, and in particular type 1 diabetes, is really suboptimal these days,” said Ashleigh Palmer, co-founder and CEO. from ProventionBio. “What we do is wait for the symptoms of the disease to present themselves to the doctors, and then the doctors treat the patient’s symptoms chronically for their entire life. The problem is that in type 1 diabetes, when the first symptoms appear, it is too late.
The treatment is presented in a single course of 14 days of infusions which each last from 30 to 60 minutes.
The most commonly reported side effects among trial participants were low white blood cells and lymph cells, skin rashes and headaches.
With type 1 diabetes, A person’s immune system attacks cells called beta cells in the pancreas that produce insulin, a hormone that helps blood sugar enter cells, where it is used for energy. The attack can occur for years before any symptoms of diabetes appear. Without insulin, blood sugar can build up in the blood and break down body fat and muscle.
Palmer says Tzield holds the disease back before symptoms appear by stopping the autoimmune disease process and the underlying destruction of beta cells. The treatment essentially reboots the immune system, preserving beta cell function.
“We really don’t have any preventative measures for type 1 diabetes to date, despite [the National Institutes of Health] funding hundreds of millions of dollars over the last 20+ years of a program called TrialNet that has tested many, many different things, including this one, and some of it came out of that work,” said said Dr. Robert Gabbay, Scientific and Scientific Director. American Diabetes Association physician. “Finally, there’s something delaying the onset of type 1 diabetes, and that’s so exciting.”
Contrary to Type 2 diabeteswhich can be prevented through lifestyle changes like weight loss and exercise, type 1 is a genetic disease that until now had no preventative options.
“For some reason, we don’t screen for type 1 diabetes, even though there are biomarkers available to show that the autoimmune disease process is already underway,” Palmer said. He added that hopes the drug will catalyze the medical system to begin population-based screening during routine child visits to intercept the disease and delay its onset.
With Tzield, doctors would screen individual family members of people with type 1 diabetes to see if they have these specific antibodies. If antibody levels are high and it looks like the person is on the verge of developing diabetes, treatment will delay this process.
“If someone has type 1, a common question that comes up is ‘well, what about my child? Will they develop type 1?’ It’s only about a 5% risk, so more often than not they won’t, but if you could find the ones that would and treat them, it can make a big difference,” Gabbay said.
A late diagnosis of type 1 diabetes could have a significant impact.
“Obviously, quality of life is significantly affected, negatively, if you’re diagnosed with type 1 diabetes. It’s a disease that never goes away,” Palmer said.
People with type 1 diabetes need to monitor their blood sugar around the clock, which affects the way they exercise and eat. High blood sugar can cause diabetic ketoacidosis, in which the body begins to break down fat for fuel and can cause a buildup of acids called ketones in the blood. This condition can lead to hospitalization, coma or death.
In 2019, about 1.9 million people had type 1 diabetes in the United States, according to the American Diabetes Association, including 244,000 children and adolescents. Type 1 affects 8% of all people with diabetes.
“The incidence of type 1 is mainly in children and adolescents, and when you’re in the turmoil of adolescence, when you just want to forget you have it,” said Olivier Bogillot, head of general medicine at Sanofi in the United States. “So when you have the ability, with treatment, to just delay the onset of disease, you can change how the quality of life is affected for families and for those children.”