Regenxbio receives FDA fast track designation for RGX-111 gene therapy for the treatment of MPS I

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Regenxbio receives FDA fast track designation for RGX-111 gene therapy for the treatment of MPS I

Regenxbio have announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-111. RGX-111 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type I (MPS I), that is designed to deliver the human iduronidase (IDUA) 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.

“Fast Track designation represents another positive step for the development of RGX-111 as we seek to address the unmet needs of people living with MPS I,”

said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO.

“We continue to engage the MPS community as we seek to provide innovative solutions to people with MPS I and their families. We look forward to working closely with the FDA to facilitate the development of RGX-111 and expect to begin the Phase I trial in the coming months.”

“There are limited treatment options that address the CNS symptoms of MPS I,”

said Steve Holland, long-time board member of the National MPS Society and father to three children with MPS I.

“Having experienced firsthand the debilitating effects that MPS I can have on children living with this condition, we are encouraged to see the FDA recognise important research that explores new treatment options for people with MPS I.”

Leading international gene therapy and lysosomal storage disease centres will participate in the Phase I clinical trial for RGX-111 for the treatment of MPS I.

Read the full press release here.

Find out more about MPS I here.