Regenxbio: FDA grants Rare Pediatric Disease Designation to RGX-181

REGENXBIO announced the U.S. 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 disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 gene. In addition to the Rare Pediatric Disease Designation, REGENXBIO previously received Orphan Drug Designation from the FDA for RGX-181. REGENXBIO plans to submit an Investigational New Drug application for RGX-181 to the FDA in the second half of 2019 to enable initiation of a first-in-human clinical trial.

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