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REGENXBIO Receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy

“REGENXBIO announced the U.S. 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.

The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. 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. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

“There is an urgent need for treatment options for CLN2 disease, a serious and life-threatening disease, which is emphasized by RGX-181 receiving Rare Pediatric Disease Designation, in addition to Orphan Drug Designation,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We believe that RGX-181 administered as a one-time treatment can potentially correct the underlying genetic condition and halt the progression of this devastating disease. We look forward to filing an IND with the FDA for the first-in-human clinical trial in the second half of 2019.””

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