BERLIN & COPENHAGEN–(BUSINESS WIRE)– 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.”
About the MuST trial, registered under NCT04729582
EEC is a rare, complex congenital malformation of the genital and urinary organs. A defined sphincter muscle defect causes urinary incontinence and can be only incompletely corrected by current treatment, which requires multiple reconstructive surgeries and lifelong management. The first-in-human trial with the lead candidate (Satori-01) targets this orphan disease and current unmet need. The phase 1/2a trial is aimed at repairing the EEC sphincter defect and will investigate both safety and efficacy.
The trial is sponsored by the Charité Universitätsmedizin and financed by the German Federal Ministry of Education and Research and the ForTra GmbH of the Else-Kröner-Fresenius Foundation.
About Orphan Drug Designation:
The Orphan Drug Act defines rare diseases as those affecting fewer than 200,000 individuals in the United States. Enacted by Congress in 1983, this legislation aims to encourage the development of medications for treating such diseases. The Orphan Drug Designation program grants orphan status to drugs and biologics intended for rare diseases that satisfy specific requirements. This designation brings along various incentives, which include tax credits for eligible clinical trials, exemption from user fees, and potential for a seven-year period of market exclusivity following approval.
About MyoPax:
MyoPax is a biotech start-up recently founded in Berlin and Copenhagen with a focus on innovative regenerative therapies for debilitating muscle disorders. It was spun out of the Charité Universitätsmedizin Berlin and the Max Delbrück Center in the Helmholtz Association and joined the BioInnovation Institute in Copenhagen with pre-seed funding. Leveraging its proprietary muscle stem cell technology and cutting-edge gene editing technologies, MyoPax aims to transform the treatment landscape and the quality of life for patients affected by muscle disorders.
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