Pharma Focus Europe

FDA Grants Rare Pediatric Disease Designation to Myopax for Breakthrough Regenerative Cell Product in Exstrophy-epispadias Complex

Tuesday, July 11, 2023

MyoPax, an innovative European biotech start-up focused on developing regenerative cell therapies for debilitating muscle disorders, has achieved a significant milestone in its efforts to help children with rare diseases. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to MyoPax's treatment for a congenital muscle defect associated with Exstrophy-Epispadias Complex (EEC). This recognition underscores the potential of MyoPax's patented technology to offer a new therapeutic approach for patients suffering from this rare and debilitating condition. Additionally, the RPDD provides regulatory and financial incentives to expedite the development process and encourage the creation of new therapies for rare pediatric diseases.

The first clinical trial for treating EEC, sponsored by Charité – Universitätsmedizin Berlin and funded by the German Federal Ministry of Education and Research and ForTra gGmbH of the Else-Kröner-Fresenius Foundation, is set to take place in Germany. Simultaneously, MyoPax is preparing for expansion into the United States to further advance their innovative treatments.

Simone Spuler, co-founder and Professor of Myology, expressed her excitement, stating, "Receiving the RPDD from the FDA is a significant milestone. It demonstrates the urgency and importance of our work in developing a transformative treatment for EEC and other muscle disorders. Currently, there are limited effective therapies available for these devastating genetic and acute conditions, and we aim to change that."

Exstrophy-Epispadias Complex (EEC) is a rare and complex congenital malformation that affects the genital and urinary organs, with a prevalence of one in 11,000 individuals. The current treatment for EEC involves multiple reconstructive surgeries and lifelong management. Unfortunately, urinary incontinence caused by a specific sphincter muscle defect can only be partially corrected.

The Rare Pediatric Disease Designation is defined by the Federal Food, Drug, and Cosmetic Act as a serious or life-threatening condition that affects individuals under the age of 18, with a prevalence of fewer than 200,000 people in the United States. Recognizing the challenges faced by pharmaceutical companies in developing treatments for these patient populations, Congress reauthorized the Creating Hope Act in 2020. This act allows companies to receive a Priority Review Voucher (PRV) upon the approval of a product with RPDD. Sponsors can utilize the PRV to expedite the priority review of a marketing application for a different product candidate or sell/transfer the voucher to another sponsor.

MyoPax's RPDD from the FDA marks a significant step forward in their mission to provide much-needed therapies to children with rare muscle disorders. By leveraging their innovative regenerative cell therapies, MyoPax aims to revolutionize the treatment landscape and bring hope to patients and their families.

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