REGENXBIO Inc. (RGNX) Tuesday said that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
The regulator has granted priority review for the BLA with a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025.
Under the partnership announced in January this year, RGX-121, upon approval, will be sold by NS Pharma, Inc., a subsidiary of Nippon Shinyaku, in the U.S.
"Acceptance of the RGX-121 BLA marks an exciting milestone on our path to bring the MPS II patient community a one-time treatment with the potential to address both the neurodevelopmental and systemic effects of Hunter syndrome," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO.
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