Thryv Therapeutics has received orphan drug designation from the FDA for LQT-1213 for the treatment of long QT syndrome

MONTREAL, Oct 1, 2024 /PRNewswire/ – Thryv Therapeutics, a clinical-stage biotech company focused on developing treatments for rare and life-threatening cardiovascular indications, today announced that the U.S. Food and Drug Administration (FDA) has approved treatment LQT-1213 orphan drug status for Long QT Syndrome (LQTS). LQT-1213 is a novel, first-in-class SGK1 inhibitor specifically designed to treat congenital LQTS. Thryv has developed a series of SGK1 inhibitors to treat cardiometabolic stress associated with various arrhythmic diseases such as LQTS, atrial fibrillation and heart failure.

“We are pleased to receive FDA orphan drug designation for LQT-1213, based in part on new positive clinical data from our Wave 1 clinical trial in patients with long QT syndrome,” he said Debra OdinkPhD, President and Chief Development Officer of Thryv Therapeutics. “People with long QT syndrome deserve properly studied and FDA-approved therapy to help them fight this potentially fatal genetic disease. There is currently no FDA-approved therapy for people with long QT syndrome. This award highlights LQT-1213's potential to address this unmet need and provides important incentive for Thryv to accelerate our efforts to conduct prospectively designed, pivotal efficacy studies in people with congenital long QT syndrome. We remain focused on providing innovative treatments for adults and children with rare diseases for whom limited options are available.”

At the FDA's request, clinical data from the ongoing Wave I clinical trial in humans with congenital LQTS were provided to the Office of Orphan Products Designation. Shortly thereafter, the company received notification that its application for orphan drug designation was approved.

FDA orphan drug designation is a special status granted to drugs and biologics used to treat rare diseases, which are diseases that affect fewer than 200,000 people in the United States. This award provides numerous benefits to support the development of treatments for rare diseases, including tax credits for clinical trial costs, exemption from certain FDA fees, and up to seven years of market exclusivity following FDA approval.