FDA Grants Orphan Drug Designation to REGENXBIO's Gene Therapy for the Treatment of Hurler's Disease

ROCKVILLE, Md., Oct. 1, 2015 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (REGENXBIO) (Nasdaq:RGNX), a leading biotechnology company in gene therapy, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to REGENXBIO's investigational gene therapy product candidate RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I).
"REGENXBIO is pleased to have received Orphan Drug Designation from the FDA for RGX-111," said Kenneth T. Mills, President and CEO of REGENXBIO. "MPS I is a severely debilitating disease and patients and their caregivers do not currently have adequate therapeutic options. We remain committed to our vision of developing gene therapies for patients with high unmet medical needs, including MPS I."
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.
MPS I is a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase (IDUA) gene. Over 1,000 individuals with MPS I are estimated to be born each year worldwide. Symptoms include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. RGX-111 uses an AAV9 vector to deliver the IDUA gene to the central nervous system.

REGENXBIO intends to file an Investigational New Drug Application (IND) with the FDA in the first half of 2016 to support the initiation of a dose-escalation Phase I/II clinical trial of RGX-111 beginning in the first half of 2016. RGX-111 is not approved for sale in the United States or elsewhere