Tolera Therapeutics granted orphan drug status for organ transplant rejection lead candidate drug

Tolera Therapeutics, Inc., was granted orphan drug status by the United States Food and Drug Administration (FDA) for its initial lead candidate, TOL101, for prophylaxis of acute rejection of solid organ transplantation.

TOL101 is a biologic protein that is designed to safely and specifically target T cells, components of the immune system that play a key role in the rejection, and ultimate failure, of transplanted organs. Organ transplantation is often the only viable treatment for many end-stage organ diseases.

"We are extremely pleased with this current action by the FDA," commented John J. Puisis, President and CEO of Tolera. "We are mobilized to bring TOL101 to market given the patient's need for safer and more effective immune suppression therapy. Doctors are in need of alternatives to traditional immune suppression approaches, and the current industry pipeline offers few solutions to advance patient care and meet this critical need."

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 therapeutic approaches.