The US Food and Drug Administration (FDA) has approved Provention Bio’s Tzield (teplizumab-mzwv) as the first and only immunomodulatory treatment to delay the onset of stage 3 type 1 diabetes in adult and paediatric patients aged eight years and older who have stage 2 type 1 diabetes.
The company submitted positive results from the randomised, double-blind, placebo-controlled TN-10 trial that included 76 patients with stage 2 type 1 diabetes.
Results showed that, after an average follow-up of 51 months, only 45% of 44 patients involved in the trial went on to develop stage 3 type 1 diabetes. All 44 patients were given Tzield intravenously once a day for two weeks.
By comparison, in the placebo group, 72% of the 32 patients were later diagnosed with stage 3 type 1 diabetes.
The mid-range time from randomisation to stage 3 type 1 diabetes diagnosis was 50 months for the Tzield-treated patient group and 25 months for those who received a placebo, representing a statistically significant delay in the development of stage 3 type 1 diabetes.
Tzield works by binding to certain immune system cells and delays the onset of stage 3 type 1 diabetes. Tzield may deactivate the immune cells that attack insulin-producing cells, while increasing the proportion of cells that help moderate the immune response.
The most common side effects of the drug include decreased levels of certain white blood cells, rash and headache.
Over 1.8 million people in the US have type 1 diabetes, an autoimmune disease that occurs when the body’s immune system attacks and destroys the cells that make insulin. People with type 1 diabetes need to inject insulin daily (or wearing an insulin pump) to survive and must check their blood sugar levels regularly throughout the day. Type 1 diabetes can occur at any age but is usually diagnosed in children and young adults. People are at higher risk if a close relative (a parent, brother or sister) has type 1 diabetes, although most patients who develop type 1 diabetes do not have a family history.
Insulin therapy and glucose monitoring are currently the standard of care for treating clinical-stage, or stage 3, type 1 diabetes, but the constant monitoring and administration of insulin represents a significant life-long burden for patients.
“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 the 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,” he added.