Regenxbio announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-121. RGX-121 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, that is designed to deliver the human iduronate 2-sulfatase (I2S) gene directly to the central nervous system (CNS) using the NAV AAV9 vector.
The FDA Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast Track-designated drugs often qualify for priority review, thereby expediting the FDA review process.
"Children living with MPS II have limited treatment options, making this Fast Track designation tremendously important," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are honored to support the MPS community and committed to offering innovative solutions to people with MPS II and their families. We look forward to working closely with the FDA to facilitate the development of RGX-121 and will begin the Phase I/II trial in the coming months."
"My family has seen firsthand the unmet needs of children with MPS II," said Jeanette Henriquez, who founded the Hunter Syndrome Foundation after her son Dominic was diagnosed with MPS II in 2011. "As we prepare to recognize International MPS Awareness Day on May 15, we are encouraged to see recognition from the FDA on important research exploring new treatment options for children with MPS II."
Leading international gene therapy and lysosomal storage disease centers will participate in the Phase I/II clinical trial for RGX-121 for the treatment of MPS II.