REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, 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.
"We believe RGX-181 has the potential to correct the underlying genetic condition, halt progression and address many of the serious and life-threatening symptoms of CLN2 disease," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "CLN2 disease is an extremely debilitating disease in children with no cure and limited treatment options. Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop RGX-181 as a potential one-time treatment for children with CLN2 disease."
FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
Read more: https://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-rgx-181-gene-therapy-for-the-treatment-of-cln2-form-of-batten-disease-300749729.html