MyoPax Receives Rare Pediatric Disease Designation from FDA for Breakthrough Regenerative Cell Product in Exstrophy-Epispadias Complex

July 10, 2023 |

2 min read

MyoPax Receives Rare Pediatric Disease Designation from FDA for Breakthrough Regenerative Cell Product in Exstrophy-Epispadias Complex.

BERLIN & COPENHAGEN, Denmark--(BUSINESS WIRE)-- The U.S. Food and Drug Administration (FDA) granted a Rare Pediatric Disease Designation (RPDD) to MyoPax (https://myopax.com), a young European biotech start-up developing innovative regenerative cell therapies for debilitating muscle disorders. The RPDD applies to a congenital muscle defect associated with Exstrophy-Epispadias Complex (EEC). This designation recognizes the potential of MyoPax’s patented technology to provide a novel therapeutic approach for patients affected by this rare, debilitating condition. The RPDD also encourages the development of new therapies for rare diseases that primarily affect children, providing regulatory and financial incentives to accelerate the development process.

The first clinical trial treating EEC (registered under NCT04729582) will be conducted in Germany under the sponsorship of Charité – Universitätsmedizin Berlin with funding from the German Federal Ministry of Education and Research and the ForTra gGmbH of the Else-Kröner-Fresenius Foundation. Meanwhile, MyoPax is preparing expansion to the US.

“Receiving the RPDD from the FDA is a significant milestone,” said Simone Spuler, co-founder and Professor of Myology. “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.”

About Exstrophy-Epispadias Complex:
EEC is a rare, complex congenital malformation of the genital and urinary organs affecting one in 11,000. Currently, treatment requires multiple reconstructive surgeries and lifelong management. Urinary incontinence is caused by a defined sphincter muscle defect and can be only incompletely corrected.

About Rare Pediatric Disease Designation:
A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act as a serious or life-threatening condition affecting individuals younger than 18 years and with a prevalence under 200,000 people in the US. Recognizing the challenges faced by drug companies in developing treatments for these patient populations, Congress reauthorized the Creating Hope Act in 2020. This act enables companies to receive a Priority Review Voucher (PRV) upon the approval of a product with a RPDD. Sponsors can use the PRV for priority review of a marketing application for a different product candidate or can sell or transfer the voucher to another sponsor.

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 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.