PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy

Core Points - The FDA has granted accelerated approval for PTC Therapeutics' gene therapy KEBILIDI™ for AADC deficiency, marking the first gene therapy approved for direct brain administration in the U.S. [1][4] - KEBILIDI is indicated for both children and adults, addressing a rare genetic disorder that severely impacts motor function due to dopamine synthesis inability [3][8]. - The therapy is delivered via a minimally invasive stereotactic neurosurgical procedure, with trained surgeons and specialized centers prepared for its administration [7][8]. Company Overview - PTC Therapeutics is focused on developing and commercializing innovative treatments for rare disorders, leveraging scientific expertise and a global commercial infrastructure [14]. - The company aims to maximize value for stakeholders by providing access to transformative medicines for patients with limited treatment options [14]. Product Details - KEBILIDI is a recombinant adeno-associated virus-based gene therapy designed to correct the genetic defect causing AADC deficiency by delivering a functioning DDC gene directly into the brain [6]. - Clinical trials have shown that KEBILIDI leads to the synthesis of dopamine and improvement in motor development milestones [3][4]. Regulatory Milestones - The approval was based on safety and efficacy findings from an ongoing global clinical trial (PTC-AADC-GT-002), with further confirmatory evidence expected from long-term patient follow-up [4]. - A Rare Disease Priority Review Voucher was also granted, which the company plans to monetize [4].