BRIEF—Regenxbio receives Rare Pediatric Disease designation for RGX-181 gene therapy

Clinical-stage US biotechnology company Regenxbio today announced the US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to RGX-181.

RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

In addition to the Rare Pediatric Disease designation, Regenxbio previously received Orphan Drug designation from the FDA for RGX-181.

If a New Biologics License application (BLA) for RGX-181 is approved, Regenxbio may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.