MyoPax has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its regenerative cell therapy in Exstrophy-Epispadias Complex (EEC). MyoPax aims to repair the congenital muscle defect associated with EEC using its cutting-edge highly regenerative muscle stem cell product.
EEC is a severe newborn condition within the uro-rectal congenital malformation spectrum impacting continence, sexual and renal function. MyoPax’s cell therapy uses patient-specific muscle stem cells, harnessing their regenerative potential through a proprietary and patented stem cell technology to repair the patient’s urinary sphincter muscle defect.
The first-in-human MuST trial is being conducted in Germany with sponsorship from the Charité Universitätsmedizin Berlin under the guidance of Prof. Simone Spuler. Prof. Wolfgang Rösch, renowned expert and pediatric urologist, will lead one of the study centers at the Hospital Barmherzige Brüder St. Hedwig in cooperation with the University of Regensburg: “This novel approach has the potential to profoundly change the treatment of Exstrophy-Epispadias Complex. We look forward to participating in the upcoming clinical trial and working together towards the goal of improving the lives of these young patients and their families.”
The FDA’s Orphan Drug Designation represents a significant milestone, reaffirming the company’s commitment to advancing innovative solutions for rare diseases. This designation provides MyoPax with several benefits, including market exclusivity and assistance with clinical trial protocols. “We are delighted to receive FDA Orphan Drug Designation for our regenerative cell therapy for Exstrophy-Epispadias Complex,” stated Dr. Verena Schöwel-Wolf, CEO of MyoPax. “This recognition validates the potential of our groundbreaking technology to address unmet medical needs and improve the lives of patients living with muscle disorders.”
