First Drug to Delay Type 1 Diabetes Approved by FDA

Last week, the Food and Drug Administration approved the first drug to delay the onset of Type 1 diabetes.

In a clinical trial of 76 participants at a high risk of diabetes, those who received the treatment were diagnosed an average of four years after the trial, compared to two years for those who received a placebo, according to NBC News’ Berkeley Lovelace Jr. In one case, the drug delayed disease onset by 11 years, the New York Times’ Gina Kolata writes.

The drug is a breakthrough in diabetes treatment, experts say, but it won’t be cheap. It’s delivered intravenously on 14 consecutive days, per Medpage Today’s Nicole Lou. Each day’s treatment will cost $13,850, for a total of $193,900. The drug, called teplizumab, should be available by the end of the year.

The FDA approved the treatment for people ages 8 and older at a high risk of developing Type 1 diabetes, according to Medscape Medical News’ Miriam E. Tucker. It cannot be used to treat Type 2 diabetes.

The approval is “really exciting” and will “turn the world of Type 1 diabetes on its head,” John Buse, a diabetes expert at the University of North Carolina who was not involved in the trial, tells the Times.

In most people, the pancreas releases insulin, a hormone that helps sugar from food travel from the bloodstream into the body’s cells, where it’s either used or stored as energy. But for people with Type 1 diabetes, the pancreas makes very little or no insulin. This causes sugar to accumulate in the bloodstream, which can lead to health complications, according to the Centers for Disease Control and Prevention (CDC). The new drug is thought to stop the immune system from attacking the insulin-making cells in the pancreas.

Close to two million people in the U.S. have Type 1 diabetes. They must monitor their blood sugar daily and take insulin injections. The American Diabetes Association estimates that people with the disease spend more than $16,000 on medical expenses annually, and about $9,600 of that is for diabetes.

Type 1 diabetes usually develops in children, teens and young adults, but it can occur at any age, according to the CDC.

Researchers recruited study participants by screening immediate family members of people with Type 1 diabetes, per the Times. Study participants had the second stage of the disease, meaning they had antibodies indicative of diabetes and abnormal blood sugar levels, but no symptoms, writes USA Today’s Adrianna Rodriguez.

The treatment is not meant to prevent people from ever developing the third and final stage of the disease, when symptoms develop. But by delaying its onset, the drug could buy people additional time that they don’t have to be treating and monitoring their diabetes, per USA Today. Almost all people with the second stage of Type 1 diabetes eventually develop the final clinical stage, and 75 percent develop it within five years, according to Medscape.

The most common side effects of the treatment were low white blood cell counts, headaches and rashes, according to Medpage Today. The drug’s label also includes a warning for cytokine release syndrome, an overly aggressive immune response to treatment.

To help with payment, one of the companies making the drug, Provention Bio, will offer a patient assistance program, a spokesperson told NBC News via email. Out-of-pocket costs will depend on patients’ insurance coverage, per the publication.

Currently, most people who are screened for early signs of Type 1 diabetes have immediate family members who have been diagnosed, per Medscape. But 85 percent of people with Type 1 diabetes don’t have a direct family member with it, according to the Times. So, in order for the treatment to reach more people, screening will have to expand.

“The most important thing right now is finding the potential patients,” Jeffrey A. Bluestone, an endocrinologist who worked on the clinical trial and has been a board member for one of the drugmakers, tells the Times.

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Will Sullivan is a science writer based in Washington, D.C. His work has appeared in Inside Science and NOVA Next.