Exstrophy-Epispadias Complex Cell Therapy Gets Orphan Drug, Rare Pediatric Disease Designations


The first-in-human MuST trial is being conducted in several centers in Germany.

The FDA has granted orphan drug designation to MyoPax’s regenerative cell therapy Satori-01 for the treatment of exstrophy-epispadias Complex (EEC).1

"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,” Prof. Dr. med. Wolfgang Rösch, pediatric urologist, Hospital BarmherzigeBrüder St. Hedwig, said in a statement.1

Satori-01 is being evaluated in the phase 1/2a first-in-human MuST trial (NCT04729582) in Germany under the guidance of Simone Spuler, MD, cofounder, MyoPax, and professor, myology, Max Delbrück Center, and Charité – Universitätsmedizin Berlin, which is sponsoring the trial. Rösch will lead one of the study centers at the hospital at the Hospital Barmherzige Brüder St. Hedwig. The trial is financed by the German Federal Ministry of Education and Research and the ForTra GmbH of the Else-Kröner-Fresenius Foundation.

READ MORE: Gene Therapy After Umbilical Cord Blood Transplantation May Help Prevent Immune Response in Patients With Krabbe Disease

"We are delighted to receive FDA Orphan Drug Designation for our regenerative cell therapy for Exstrophy-Epispadias Complex," Verena Schöwel-Wolf, MD, MBA, chief executive officer, MyoPax, added.1 "This recognition validates the potential of our groundbreaking technology to address unmet medical needs and improve the lives of patients living with muscle disorders."

EEC is a rare congenital disease that causes malformation of the genital and urinary organs which includes a defined sphincter muscle defect. The defect impacts urinary continence, sexual, and renal function, and current standard of care entails multiple reconstructive surgeries and lifelong management which can only achieve an incomplete correction. Satori-01 consists of patient-specific muscle stem cells and is designed to repair the urinary sphincter muscle defect associated with EEC. The MuST trial will evaluate safety and efficacy of Satori-01 in patients with EEC. The orphan drug designation follows the rare pediatric disease designation the FDA granted to MyoPax earlier in July for the same program.2

"Receiving the RPDD from the FDA is a significant milestone,” Spuler said in a statement at that time.2 “It reflects the urgency and importance of our work in developing a transformative treatment for EEC and beyond. Muscle disorders encompass many devastating genetic and acute diseases for which today there are only supportive therapies.”

1. MyoPax receives FDA orphan drug designation for its innovative regenerative cell therapy for exstrophy-epispadias complex. News release. MyoPax. July 26, 2023. https://www.businesswire.com/news/home/20230726215606/en/MyoPax-Receives-FDA-Orphan-Drug-Designation-for-its-Innovative-Regenerative-Cell-Therapy-for-Exstrophy-Epispadias-Complex
2. MyoPax receives rare pediatric disease designation from FDA for breakthrough regenerative cell product in exstrophy-epispadias complex. News release. MyoPax. July 10, 2023. https://www.businesswire.com/news/home/20230710526334/en/MyoPax-Receives-Rare-Pediatric-Disease-Designation-from-FDA-for-Breakthrough-Regenerative-Cell-Product-in-Exstrophy-Epispadias-Complex
Related Videos
Jeffrey Chamberlain, PhD
Robert J. Hopkin, MD
Alan Beggs, PhD
Paul Harmatz, MD
Paul Harmatz, MD
Heather Lau, MD, MS, the executive director of global clinical development at Ultragenyx Pharmaceutical
Heather Lau, MD, MS, the executive director of global clinical development at Ultragenyx Pharmaceutical
Paul Harmatz, MD, on Challenges With Assessing Neurocognitive Outcomes
Paul Harmatz, MD, on Assessing D2S6 in Trials for MPS Type 2
© 2024 MJH Life Sciences

All rights reserved.