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Myopax's Novel Regenerative Cell Therapy for Exstrophy-Epispadias Complex Granted FDA Orphan Drug Designation

Thursday, July 27, 2023

MyoPax, a biotechnology company, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its advanced regenerative cell therapy aimed at treating Exstrophy-Epispadias Complex (EEC). EEC is a rare congenital condition within the uro-rectal malformation spectrum that severely impacts continence, sexual function, and renal function in newborns. MyoPax's innovative therapy utilizes patient-specific muscle stem cells and a proprietary patented stem cell technology to repair the defective urinary sphincter muscles associated with EEC.

The groundbreaking MuST trial is the first of its kind and is taking place in Germany under the guidance of Prof. Simone Spuler from Charité Universitätsmedizin Berlin. The trial, led by renowned pediatric urologist Prof. Wolfgang Rösch, will be conducted at the Hospital Barmherzige Brüder St. Hedwig in collaboration with the University of Regensburg. The primary objective of the trial is to assess the safety and efficacy of this novel approach in treating Exstrophy-Epispadias Complex and to potentially enhance the quality of life for affected patients and their families.

The FDA's granting of Orphan Drug Designation is a significant achievement for MyoPax, affirming their dedication to developing innovative solutions for rare diseases. This designation offers several advantages, including market exclusivity and assistance in designing clinical trial protocols. Dr. Verena Schöwel-Wolf, CEO of MyoPax, expressed her excitement about the FDA Orphan Drug Designation, emphasizing the validation of their groundbreaking technology's potential to address unmet medical needs in muscle disorders.

EEC presents as a complex and rare congenital malformation of the genital and urinary organs, leading to urinary incontinence due to a specific sphincter muscle defect. Current treatments are only partially effective, necessitating multiple reconstructive surgeries and lifelong management. MyoPax's lead candidate, Satori-01, will be evaluated in the phase 1/2a MuST trial to target this orphan disease and address the existing unmet medical needs by repairing the sphincter defect and assessing safety and efficacy.

The MuST trial is sponsored by Charité Universitätsmedizin and funded by the German Federal Ministry of Education and Research and the ForTra GmbH of the Else-Kröner-Fresenius Foundation.

The Orphan Drug Act, passed by Congress in 1983, defines rare diseases as those affecting fewer than 200,000 individuals in the United States. This legislation aims to encourage the development of medications for such diseases. Orphan Drug Designation is granted to drugs and biologics intended for rare diseases that meet specific requirements. It provides various incentives, including tax credits for eligible clinical trials, exemption from user fees, and the potential for a seven-year period of market exclusivity after approval.

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