True North Therapeutics, a clinical stage biotechnology company developing novel product candidates that selectively inhibit the Complement system to treat certain rare diseases, has announced that the FDA has granted Orphan Drug Designation for the company’s lead product candidate TNT009, for the treatment of autoimmune hemolytic anemia, including Cold Agglutinin Disease (CAD), a form of autoimmune hemolytic anemia for which there are limited treatment options available for patients.
TNT009 is currently in a phase Ib clinical study in patients with CAD, and positive interim results from the study were announced in June 2016. True North also received Orphan Drug Designation for TNT009 from the EMA in February 2016.
“Receiving both U.S. and EMA Orphan Drug Designation for TNT009 is an important milestone in the development of much needed new treatment options for patients with CAD,” said Nancy Stagliano, Ph.D., chief executive officer of True North. “We look forward to continuing development of TNT009 for CAD and reporting additional phase Ib clinical data when available.”
Orphan drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity.