MyoPax Scores Major FDA Approval for Revolutionary Cell Therapy to Treat Exstrophy-Epispadias Complex

MyoPax has achieved a major breakthrough in Exstrophy-Epispadias Complex (EEC) treatment, with the U.S. Food and Drug Administration (FDA) granting it Orphan Drug Designation. The revolutionary regenerative cell therapy developed by MyoPax is specifically designed to repair the congenital muscle defect of EEC patients using its cutting-edge muscle stem cell product. This treatment offers unprecedented hope to EEC sufferers and their families.

MyoPax’s groundbreaking cell therapy offers a potential solution to the life-altering conditions caused by uro-rectal congenital malformations in newborns. This patient-specific muscle stem cell therapy is designed to restore functioning of the urinary sphincter muscle, improving continence, sexual and renal function. Newly developed and patented stem cell technology is used to harness the regenerative power of these stem cells to improve the patient’s condition.

The Charité Universitätsmedizin Berlin, under the guidance of Prof. Simone Spuler, is conducting the groundbreaking MuST trial – the first of its kind – in Germany. led by Prof. Wolfgang Rösch, the renowned pediatric urologist and expert, the trial will have a study center at the Hospital Barmherzige Brüder St. Hedwig in collaboration with the University of Regensburg.

There is genuine optimism that this new approach can dramatically enhance the treatment of Exstrophy-Epispadias Complex, improving the lives of patients and their families alike. Prof. Rösch enthusiastically remarks, “We look forward to participating in the upcoming clinical trial and working together towards this common goal.”

MyoPax is thrilled to announce the recent FDA Orphan Drug Designation for its regenerative cell therapy for Exstrophy-Epispadias Complex. This revolutionary new therapy promises to revolutionize treatment for patients living with muscle disorders – a cause the company is deeply passionate about.

The designation provides numerous benefits, including invaluable assistance with clinical trial protocols and exclusive market access. As CEO Dr. Verena Schöwel-Wolf shared, “We look forward to further exploring the potential of our innovative technology and bringing these much needed treatments closer to thousands of patients.”

About the MuST trial, registered under NCT04729582

The revolutionary new Satori-01 candidate aims to bring much needed relief to those suffering from the rare and complex congenital malformation known as EEC. By addressing the severe sphincter muscle defect that causes urinary incontinence, the phase 1/2a trial hopes to repair the defect and improve the lives of those with this debilitating condition.

With multiple reconstructive surgeries and lifelong management generally required for current treatments, the first-in-human trial is a beacon of hope for those waiting for a cure.

The Charité Universitätsmedizin have sponsored a trial that is proudly funded by the German Federal Ministry of Education and Research and the ForTra GmbH of the Else-Kröner-Fresenius Foundation. Join us in our mission to make discoveries that will shape the future of healthcare!

About Orphan Drug Designation:

The Orphan Drug Act of 1983 was put in place to promote the development of treatments for rare diseases that affect less than 200,000 individuals in the United States. To encourage this, the Orphan Drug Designation program grants pharmaceuticals and biologics the orphan status through meeting certain conditions.

The incentives for obtaining orphan status include tax credits for eligible clinical trials, exemption from user fees, and a potential seven-year period of market exclusivity following approval.

About MyoPax:

MyoPax is an ambitious biotech start-up with a mission to revolutionize the treatment of debilitating muscle disorders. Founded in Berlin and Copenhagen, the company was created in collaboration between Charité Universitätsmedizin Berlin and Max Delbrück Center in the Helmholtz Association, and joined the BioInnovation Institute in Copenhagen with pre-seed funding. MyoPax harnesses stem cell technology and gene editing techniques to tackle muscle disorders, aiming to improve the quality of life for those affected by this condition.

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