Anticipating a breakthrough therapy for type 1 diabetes

The Specialty Pharmacy Pipeline Drugs to Watch Report for Q3-Q4 2022 highlights a new drug, teplizumab (Provention Bio), to delay onset of clinical type 1 diabetes (T1D) in at-risk individuals ages 8 to 45 years. In a Phase II trial, after an outpatient intravenous course of teplizumab over two weeks, the median time to clinical diagnosis of T1D in high-risk patients was five years compared to two years for the placebo group. The drug is pending FDA approval in November 2022.

Type 1 diabetes is an autoimmune disorder that results from the loss of beta cells. The absence of these cells means the pancreas does not make insulin or produces an insufficient amount of insulin. Insulin is a hormone that helps blood glucose (sugar) enter the cells of the body where it can be used for energy. Without insulin, blood sugar can’t enter the cells and builds up in the bloodstream. High blood sugar is damaging to the body and causes many of the symptoms and complications of diabetes.¹

Type 1 diabetes affects approximately 1.9 million people in the United States.² Additionally, an estimated 300,000 are in the early states of the disease and show no symptoms.³ These patients are said to have presymptomatic T1D, and approximately 100,000 to 150,000 of them are considered to be at high risk for progressing to symptomatic (insulin dependent) T1D.⁴

Type 1 diabetes is one of only two major autoimmune diseases without any approved disease-modifying therapy.⁵ Teplizumab delays the onset of T1D by slowing the destruction of beta calls. It was granted Breakthrough Therapy designation and would be the first approved therapy to delay T1D.

The report details anticipated impact on spend for this therapy. Teplizumab may result in some medical cost avoidance due to being a preventive therapy and thus delaying cost of active disease treatment (e.g., insulin therapy).

The report also includes planned updates to CVS Health’s Specialty Guideline Management.