FDA on Thursday approved the first treatment designed to delay the onset of insulin-dependent Type 1 diabetes. And while the drug could provide millions of at-risk "patients with months to years without the burdens of disease," some experts are highlighting potential challenges with prescribing the drug.

FDA approves 'an important new treatment' for Type 1 diabetes  

ProventionBio on Thursday announced that FDA approved teplizumab—a monoclonal antibody that delayed the onset of insulin-dependent Type 1 diabetes in clinical trials involving patients with autoantibody markers of early risk. In the trials, the treatment delayed onset of disease by over two years in some patients.

The treatment, which will be sold under the brand name Tzield by pharmaceutical companies ProventionBio and Sanofi, is administered through intravenous infusion.

FDA approved the treatment for adults and children ages 8 and older with stage 2 Type 1 diabetes. Teplizumab is designed to slow the body's attack on the cells that produce insulin, giving patients more time before they have to rely on pharmaceutical insulin. Individuals who have insulin-dependent Type 1 diabetes, pre-Type 2 diabetes, or Type 2 diabetes are not eligible for the treatment.

According to Provention, the treatment will cost $13,850 per vial or $193,900 for a full 14-day treatment. "We expect to have drug in the channel by the end of the year," said Jason Hoitt, Provention's CCO, noting that the company is currently working with Sanofi toward a full launch in January 2023.

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Many experts have underscored the impact of a delayed diagnosis of insulin-dependent Type 1 diabetes.

"Obviously, the quality of life is substantially impacted, negatively impacted, if you are diagnosed with type 1 diabetes. It's a disease that never goes away," said ProventionBio co-founder and CEO Ashleigh Palmer.

According to the American Diabetes Association (ADA), roughly 1.9 million Americans had Type 1 diabetes as of 2019, including 244,000 children and adolescents. Each year, around 64,000 Americans are diagnosed with insulin-dependent Type 1 diabetes, according to CDC.

"The incidence of the type 1 is mainly in kids and teenagers, and when you are in the turmoil of adolescence, when you just want to forget that you have it," said Olivier Bogillot, Sanofi's head of general medicines in the United States. "So when you have the ability with a treatment to just delay the onset of the disease, you can change the way the quality of life is impacted for families and for those kids."

"Today's approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients," said John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity in FDA's Center for Drug Evaluation and Research. "The drug's potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease."

"This approval is a watershed moment for the treatment and prevention of type 1 diabetes," said Mark Anderson, director of the University of California San Francisco Diabetes Center. "Until now, the only real therapy for patients has been a lifetime of insulin replacement. This new therapy targets and helps to halt the autoimmune process that leads to the loss of insulin."

"We really have no preventative measure for type 1 diabetes to date, and that is despite [the National Institutes of Health] funding hundreds of millions of dollars over the last 20-plus years of a program called TrialNet that has tested many, many different things, including this, and some of this came out of that work," said Robert Gabbay, ADA's chief scientific and medical officer. "Finally, there is something that delays the onset of type 1 diabetes, and it's so exciting."

Experts encourage 'general population screening' for Type 1 diabetes

Individuals with a family history of Type 1 diabetes or concerns about developing the disease can get a blood test to detect autoantibodies—diagnostic markers that have been shown to predict insulin dependence. However, screening for the disease is not currently part of routine care.

"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, adding that she hopes the drug will promote population-based screening during routine well visits to detect the disease and delay its onset in at-risk individuals.

According to Aaron Kowalski, CEO of JDRF, the primary challenge in prescribing teplizumab will be identifying who needs it.

"Screening becomes a really big issue, because what we know is, about 85% of type 1 diagnoses today are in families that don't have a known family history," Kowalski said. "Our goal is to do general population screening" with blood tests to look for markers of the disease.

"The way in which not just industry but our medical system go about managing autoimmune diseases, and especial type 1 diabetes, is really suboptimal in today's day and age," Palmer added. "What we do is, we wait until the symptoms of the disease present to doctors, and then doctors treat the patient's symptoms chronically for a lifetime. The trouble is that in type 1 diabetes, when the symptoms first present, it's too late." (Christensen/Goodman, CNN, 11/18; Strauss/Winsor, ABC News, 11/18; Satija, Reuters, 11/17; Kolata, New York Times, 11/18)