The U.S. Food and Drug Administration (FDA) has approved the first disease-modifying therapy for type 1 diabetes (T1D) that delays the onset of disease in certain age groups. Biopharmaceutical company Provention Bio with its monoclonal antibody teplizumab, branded under the name Tzield, has received the go-ahead.
“Today’s approval of a first-in-class therapy adds an important new treatment option for certain at-risk patients,” says John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity in the FDA’s Center for Drug Evaluation and Research in a statement by the FDA.
“The drug’s potential to delay clinical diagnosis of type 1 diabetes may provide patients with months to years without the burdens of disease.”
T1D occurs when a person's own immune system attacks and destroys specialized cells in the pancreas that produce insulin, the hormone that regulates blood sugar levels.
The disease normally progresses in stages over time as more of the pancreatic cells are destroyed. Stage 1 is the first sign of the disease. Stage 2 is the presence of β-cell autoimmunity with dysregulation of blood sugar levels, but patients are still normally presymptomatic at this point. Stage 3 is the advanced onset of the symptomatic disease, which normally becomes irreversible at this point.
T1D could occur in anyone at any age – however, it's normally first diagnosed in children and young people. When this happens, individuals are normally required to take insulin injections or use an insulin pump for the rest of their lives to manage their blood sugar levels to avoid life-threatening complications.
"The progression of T1D can be particularly onerous; patients who progress from Stage 2 to Stage 3 T1D can develop diabetic ketoacidosis, which can be life threatening and is experienced by up to 50% of Stage 3 patients at the time of presentation," said Dr Eleanor Ramos, Chief Medical Officer at Provention Bio, in a statement. "The onset of Stage 3 T1D is a life-changing moment – once insulin-producing cells are no longer capable of maintaining normal glycemic control, this irreversible condition can lead to the need, in just one year, for a patient, 1,460 finger sticks to check blood glucose levels, around 1,100 insulin injections, and experiencing an average of 127 episodes of hypoglycemia. These complications can cause stress, fear, and anxiety in patients as they work to manage their T1D diagnosis and provide perspective on the meaning of a delay in the onset of Stage 3 T1D."
In clinical trials, Tzield showed effectiveness at delaying the onset T1D from stage 2 to stage 3, the point where the symptomatic disease normally begins. The safety and efficacy of the drug were evaluated In the randomized, double-blinded trial with two groups of patients with stage 2 diabetes. One group was randomly selected to receive the monoclonal antibody teplizumab, and the other a placebo. Those that received teplizumab had a delayed onset from stage 2 to stage 3 T1D by over two years and showed benefit compared to patients that had only received the placebo.
"This is a historic occasion for the T1D community and a paradigm shifting breakthrough for individuals aged 8 years and older with Stage 2 T1D who now have a therapy approved by the FDA to delay the onset of Stage 3 disease. It cannot be emphasized enough how precious a delay in the onset of Stage 3 T1D can be from a patient and family perspective; more time to live without and, when necessary, prepare for the burdens, complications and risks associated with Stage 3 disease" concludes Ashleigh Palmer, Co-Founder and CEO of Provention Bio in a statement.
For more information, visit the FDA's announcement.