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FDA Grants Rare Pediatric Disease Designation for MyoPax’ Regenerative Cell Product

July 10, 2023

FDA Grants Rare Pediatric Disease Designation for MyoPax’ Regenerative Cell Product in Exstrophy-Epispadias Complex

Rare Daily Staff

The U.S. Food and Drug Administration granted MyoPax a Rare Pediatric Disease designation to for its regenerative cell therapy for a congenital muscle defect associated with exstrophy-epispadias complex.

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.

The first clinical trial treating EEC 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 United States.

“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.”

RPDD recognizes the potential of MyoPax’s patented technology to provide a novel therapeutic approach for patients affected by this rare, debilitating condition. The Rare Pediatric Disease designation also encourages the development of new therapies for rare diseases that primarily affect children, providing regulatory and financial incentives to accelerate the development process.

A rare pediatric disease is defined as a serious or life-threatening condition affecting individuals younger than 18 years and with a prevalence under 200,000 people in the United States. Recognizing the challenges faced by drug companies in developing treatments for these patient populations. This act enables companies to receive a Priority Review Voucher upon the approval of a product with a Rare Pediatric Disease designation. 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. Most recently, Sarepta Therapeutics sold a PRV for $102 million.

Photo: Simone Spuler, co-founder and professor of Myology

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