ClearPoint congratulates partner PTC Therapeutics on completion of its BLA

ClearPoint Neuron congratulates its partner PTC Therapeutics on completion of its BLA submission to the U.S. Food and Drug Administration, FDA, for the approval of Upstaza, an investigational treatment for AADC Deficiency. If approved, Upstaza would become the first therapy to treat AADC Deficiency in the United States. “AADC Deficiency is a devastating rare pediatric movement disorder that causes significant developmental delays and autonomic symptoms starting from birth. Patients with AADC deficiency are at a high risk of death in the first decade of life,” stated Jeremy Stigall, Chief Business Officer at ClearPoint Neuro. “The Upstaza(TM) BLA is the first filing for FDA approval of a treatment that addresses this devastating condition. Through our partnership with PTC, we are demonstrating our commitment to drive progress for the AADC Deficiency community.”