We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA’s accelerated approval pathway for the treatment of Hunter Syndrome, Mucopolysaccharidosis Type II (MPS II). The company also announced that a pivotal program for RGX-121 is active and enrolling patients.
RGX-121 is an investigational, one-time AAV Therapeutic that delivers the gene that encodes the iduronate-2-sulfatase (I2S) enzyme that MPS II patients are missing to break down cellular waste. Their ongoing trial of RGX-121 has been expanded into a pivotal phase trial, called CAMPSIITE™ which is now expected to enroll up to 10 MPS II patients through early 2023, with the potential to enroll additional patients after this achievement.
This brings another potential gene therapy for MPS II in an exciting time of new developments in treatment.