PARAMUS,
N.J., May 14, 2025 /PRNewswire/ -- NS Pharma,
Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon
Shinyaku) announced today that the U.S. Food and Drug
Administration has accepted for review the Biologics License
Application (BLA) submission by REGENXBIO Inc. (REGENXBIO;
Headquarters: Rockville, Maryland,
USA; CEO: Curran M. Simpson, NASDAQ:
RGNX) for RGX-121 (clemidsogene lanparvovec), a potential
first-in-class, investigational gene therapy for the treatment of
Mucopolysaccharidosis II (MPS II). The FDA granted REGENXBIO's
BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA")
target action date of November 9,
2025.
In January 2025, Nippon Shinyaku
and REGENXBIO entered into a strategic partnership for the
development and commercialization of RGX-121, as well as RGX-111,
which is for the treatment of MPS I. Upon potential approval of
RGX-121, NS Pharma will be exclusively responsible for
commercializing RGX-121 in the U.S.
"This FDA decision represents a significant milestone in
bringing a new, potentially life-changing treatment option to
patients in the MPS community," said NS Pharma President, Yukiteru
Sugiyama, Ph.D. "We are excited about our partnership with
REGENXBIO and the value of our combined expertise in generating
renewed hope for MPS families."
For more details, please see the press release from REGENXBIO:
https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-fda-acceptance-and-priority-review-bla-rgx
About RGX-121 (clemidsogene
lanparvovec)
RGX-121 is a potential one-time AAV
therapeutic for the treatment of boys with MPS II, designed to
deliver the iduronate-2-sulfatase (IDS) gene to the central nervous
system (CNS). Delivery of the IDS gene within cells in the CNS
could provide a permanent source of secreted iduronate-2-sulfatase
(I2S) protein beyond the blood-brain barrier, allowing for
long-term cross correction of cells throughout the CNS. RGX-121
expressed protein is structurally identical to normal I2S.
RGX-121 has received Orphan Drug Product, Rare Pediatric
Disease, Fast Track and Regenerative Medicine Advanced Therapy
designations from the FDA.
About MPS II
MPS II, or Hunter Syndrome, is a
rare, X-linked recessive disease caused by a deficiency in the
lysosomal enzyme I2S leading to an accumulation of
glycosaminoglycans (GAGs), including heparan sulfate (HS) in
tissues which ultimately results in cell, tissue, and organ
dysfunction, including in the CNS. In severe forms of the disease,
early developmental milestones may be met, but developmental delay
is readily apparent by 18 to 24 months. Specific treatment to
address the neurological manifestations of MPS II remains a
significant unmet medical need. Key biomarkers of I2S enzymatic
activity in MPS II patients include its substrate heparan sulfate
(HS) D2S6, which has been shown to correlate with neurocognitive
manifestations of the disorder.
About REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. Since its founding
in 2009, REGENXBIO has pioneered the development of AAV
Therapeutics, an innovative class of gene therapy medicines. For
more information, please visit www.regenxbio.com.
About NS Pharma, Inc.
NS Pharma, Inc., is a wholly
owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a
registered trademark of the Nippon Shinyaku Co., Ltd. For more
information, please visit nspharma.com.
US Media Contact:
media@nspharma.com
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SOURCE NS Pharma, Inc.