MyoPax receives FDA Orphan Drug Designation

We are proud to announce that MyoPax has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for its innovative regenerative cell therapy for Exstrophy-Epispadias-Complex.


After recently receiving the FDA’s Rare Pediatric Disease Designation, this marks the next pivotal milestone in our quest to restore muscle function and improve quality of life for those facing this congenital muscle defect.

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.

Learn more about the first-in-human MuST: