01 December 2022

Immunosuppressor against diabetes

The FDA has approved the world's first drug for the prevention of type one diabetes

Sergey Zadvoryev, N+1

American Food and Drug Administration (FDA) approved the world's first drug for the prevention of type I diabetes mellitus in children and adults. Teplizumab, a recombinant drug based on monoclonal antibodies against the CD3 lymphocytic receptor, became the first drug for the prevention of type I diabetes.

Type I diabetes mellitus is an autoimmune disease. It usually occurs in childhood or adolescence. The immune system of predisposed people, when confronted with certain antigens, forms an immune response against beta cells of the pancreatic islets that produce insulin. At this stage of pathogenesis, the disease is asymptomatic, although there are already autoantibodies in the blood.

As beta cells are damaged, subclinical disorders of carbohydrate metabolism form over several years (this is the second stage of pathogenesis), which can be detected during routine examination. But patients usually come under medical supervision only at the third stage of pathogenesis, when symptoms of diabetes appear.

By this time, there are almost no insulin-producing cells left in the pancreatic islets, and the patient can be effectively helped by giving insulin for life (for example, with the help of insulin pumps, which are becoming more and more perfect) or by transplanting beta cells (everything is not so successful here yet).

But you can try to prevent the disease by suppressing autoimmune reactions in the early stages of pathogenesis. Over the past 20 years, scientists have studied several immunosuppressants as a means of preventing type I diabetes, of which teplizumab is the most widely studied. It is a recombinant monoclonal antibody against CD3, the surface receptor of T-lymphocytes. After CD3 blockade, the phenotype of lymphocytes changes: they produce cytokines worse and proliferate worse in response to the antigen. But there have been no immunosuppressants registered in the world for the prevention of type I diabetes.

On November 17, the FDA registered teplizumab for the prevention of type I diabetes mellitus in children from the age of eight and in adults with the second stage of the pathogenesis of type I diabetes mellitus. The medicine is intended for intravenous administration, the course of treatment lasts two weeks. The cost of such a course, based on the statement of the representatives of the manufacturer, will be about two hundred thousand dollars.

The medicine has proven its effectiveness, but it is not one hundred percent. In a study with a four-year follow-up period, teplizumab reduced the risk of clinically pronounced diabetes by 2.5 times. Judging by individual seven-year observations, one course of treatment delays the manifestation of the disease for 2-3 years.

Teplizumab has a long history of entering the market. The compound was synthesized in the 1990s. In 2007-2011, the pharmaceutical company Eli Lilly tried to use it to slow down the progression of type I diabetes that had already occurred (at the third stage of the pathogenesis of the disease), but failed.

Six years later, Provision Bio acquired a license for teplizumab and, after conducting research, tried to register in 2021 — this time as a means to prevent diabetes. The FDA panel of Experts recognized that the benefits of the treatment outweighed the risks and recommended approval of the application. But a month later, the regulator demanded additional studies of the bioequivalence of teplizumabs produced by different companies on different equipment throughout the history of drug research. Additional studies were completed by March, and now the drug has finally been registered, becoming the first means of delaying type I diabetes.

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