FDA Grants Rare Pediatric Disease Designation to MavriX Bio
The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to MavriX Bio for its investigational gene therapy, MVX-220, for the treatment of Angelman syndrome.
MIDDLETON, Massachusetts – MavriX Bio announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational gene therapy, MVX-220, for the treatment of Angelman syndrome (AS). AS is a rare and debilitating neurogenetic disorder for which no approved treatments currently exist.
The designation is considered an important milestone for the company's development program. Angelman syndrome is caused by the loss of function of the maternally inherited Ubiquitin Protein Ligase E3A (UBE3A) gene. MVX-220 is an investigational adeno-associated virus (AAV)-based gene therapy designed to deliver a functional copy of the UBE3A gene to neurons, aiming to address the underlying genetic cause of the disease.
The RPDD provides incentives for the development of therapies for rare pediatric diseases. If MVX-220 receives FDA approval and meets statutory requirements, the program may be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). This voucher can be used to obtain priority review of a subsequent marketing application or sold to another sponsor.
MVX-220's development was advanced with funding from the Foundation for Angelman Syndrome Therapeutics (FAST) and is progressing in the first-in-human ASCEND-AS study (NCT07181837), which is evaluating the safety and tolerability of the therapy in adult and pediatric individuals with AS.