Provention Bio announced the U.S. FDA granted Breakthrough Therapy Designation (BTD) to teplizumab (PRV-031) for the prevention or delay of clinical type 1 diabetes (T1D) in individuals at-risk of developing the disease.
BTD is an FDA program designed to expedite the development and review of therapeutic candidates intended to treat serious or life-threatening diseases. To qualify for this designation, preliminary clinical evidence must indicate that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. The benefits to Provention of this BTD include more intensive and interactive guidance from FDA on an efficient drug development program, access to a scientific liaison to help expedite review time, and eligibility for Priority Review if relevant criteria are met.
BTD status was granted based on clinical data from the “At-Risk” Study conducted by TrialNet, which showed that a single 14-day course of PRV-031 (teplizumab) significantly delayed the onset and diagnosis of clinical T1D, as compared to placebo, by a median of at least 2 years in children and adults considered to be at high risk of developing clinical T1D.
Provention is currently evaluating PRV-031 in patients newly diagnosed with clinical T1D in the Phase 3 PROTECT Study.
About Type 1 Diabetes
1D is an autoimmune disease characterized by the immune system’s attack upon the body’s own insulin-producing pancreatic beta cells by a type of white blood cell known as a self-reactive T cell. At the same time, another type of white blood cell known as a regulatory T cell, which normally controls the self-reactive T cells, is unable to suppress the immune system’s attack. Individuals with T1D require a life-long dependence on insulin products delivered through multiple daily injections, continuous infusion pumps, or other delivery methods.
About teplizumab (PRV-031)
Monoclonal antibodies (mAbs) are man-made immune proteins used to treat various diseases. PRV-031 is a humanized mAb that binds with high specificity to a cell surface protein on T cells called CD3 (cluster of differentiation 3). The CD3 protein is a co-receptor that participates in the activation of T cells during the immune response.
Experimental data suggest that the binding of PRV-031 to CD3 triggers events that differentially inhibit the activation of self-reactive T cells, without affecting regulatory T cells. This restores the important state of immune tolerance and may prevent self-reactive T cells from attacking beta cells in the pancreas. If administered shortly after T1D diagnosis, PRV-031 has the potential to intercept the T1D disease process and slow or prevent the complete destruction of insulin-producing pancreatic beta cells.