FDA grants Tolera's TOL101 orphan drug designation for treatment of T1DM

Tolera Therapeutics, Inc., was granted orphan drug status by the United States Food and Drug Administration (FDA) for TOL101 (anti-TCR murine monoclonal antibody, type IgM), for treatment of recent onset immune-mediated Type 1 diabetes mellitus (T1DM).  Designation is granted for treatment of patients sixteen years of age and younger with immune-mediated T1DM and preserved pancreatic beta-cell function, commonly referred to as juvenile diabetes.

"We are pleased with this designation," commented John J. Puisis, President and CEO of Tolera.  "Clinicians and patients need an effective and safe means to modulate the immune system, particularly for juvenile diabetes patients.  Current treatments and industry pipelines offer very few solutions to advance patient care, leaving these young patients with a deteriorating disease condition.  Thus far, these treatments can only offset disease symptoms, or slow disease progression, but at the cost of potentially serious side effects. We believe we are developing a therapy that will prove to be both safe and effective, thus providing the patient with a much better treatment outcome."

The Orphan Drug Act was enacted to allow the FDA to grant a product special orphan status when it is intended to treat rare diseases or conditions -- those that affect fewer than 200,000 people annually in the United States. This special designation waives governmental filing fees, provides the drug sponsor with tax credits related to development expenses, as well as other support.  In addition, orphan drug designation creates the opportunity for the sponsor to apply for orphan drug grant money from the U.S. government to help support clinical trials.  As an agent with the potential to aid patients with a severe condition, TOL101 meets the stringent FDA requirements for orphan drug designation.  This designation will facilitate TOL101's entry into the clinic, where relatively few new therapies have emerged over the last ten years, and there is a growing need for safer, more targeted immunological therapeutic approaches.