- FDA assigns PDUFA target action date of November 9, 2025
- RGX-121 on track to be the first gene therapy and one-time
treatment for MPS II
- Partner Nippon Shinyaku to lead commercialization upon
potential approval
- REGENXBIO to lead commercial manufacturing and supply
chain
ROCKVILLE, Md., May 13, 2025
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
the U.S. Food and Drug Administration (FDA) has accepted for review
the Biologics License Application (BLA) seeking accelerated
approval for clemidsogene lanparvovec (RGX-121) for the treatment
of Mucopolysaccharidosis II (MPS II), also known as Hunter
syndrome.
The FDA granted the BLA Priority Review with a Prescription Drug
User Fee Act (PDUFA) target action date of November 9, 2025.
"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. "Supported by positive biomarker data
and long-term outcomes, RGX-121 has the potential to be a
first-in-class gene therapy that could dramatically transform the
MPS II treatment landscape and reduce the significant burden
patients and families currently face with weekly enzyme replacement
therapy."
RGX-121 has received Orphan Drug Product, Rare Pediatric
Disease, Fast Track and Regenerative Medicine Advanced Therapy
(RMAT) designations from the U.S. Food and Drug
Administration and advanced therapy medicinal products (ATMP)
classification from the European Medicines Agency.
Under the strategic partnership announced in January 2025, following potential FDA approval,
RGX-121 will be commercialized by NS Pharma, Inc., a wholly-owned
subsidiary of Nippon Shinyaku, in the U.S. Approval of RGX-121
could result in receipt of a Priority Review Voucher (PRV).
REGENXBIO retains all rights to, and 100 percent of any proceeds
related to the potential sale of, the PRV for RGX-121.
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.
About Mucopolysaccharidosis Type II (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 biotechnology
company on a mission to improve lives through the curative
potential of gene therapy. Since its founding in 2009, REGENXBIO
has pioneered the field of AAV gene therapy. REGENXBIO is advancing
a late-stage pipeline of one-time treatments for rare and retinal
diseases, including RGX-202 for the treatment of Duchenne;
clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and
RGX-111 for the treatment of MPS I, both in partnership with Nippon
Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the
treatment of wet AMD and diabetic retinopathy, in collaboration
with AbbVie. Thousands of patients have been treated with
REGENXBIO's AAV platform, including those receiving Novartis'
ZOLGENSMA®. REGENXBIO's investigational gene therapies have the
potential to change the way healthcare is delivered for millions of
people. For more information, please
visit www.REGENXBIO.com.
FORWARD-LOOKING STATEMENTS
This press release includes
"forward-looking statements," within the meaning of Section 27A of
the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended. These statements
express a belief, expectation or intention and are generally
accompanied by words that convey projected future events or
outcomes such as "believe," "may," "will," "estimate," "continue,"
"anticipate," "assume," "design," "intend," "expect," "could,"
"plan," "potential," "predict," "seek," "should," "would" or by
variations of such words or by similar expressions. The
forward-looking statements include statements relating to, among
other things, REGENXBIO's future operations, clinical trials, costs
and cash flow. REGENXBIO has based these forward-looking statements
on its current expectations and assumptions and analyses made by
REGENXBIO in light of its experience and its perception of
historical trends, current conditions and expected future
developments, as well as other factors REGENXBIO believes are
appropriate under the circumstances. However, whether actual
results and developments will conform with REGENXBIO's expectations
and predictions is subject to a number of risks and uncertainties,
including the timing of enrollment, commencement and completion and
the success of clinical trials conducted by REGENXBIO, its
licensees and its partners, the timing of commencement and
completion and the success of preclinical studies conducted by
REGENXBIO and its development partners, the timing or likelihood of
payments from AbbVie or Nippon Shinyaku, the monetization of any
priority review voucher, the timely development and launch of new
products, the ability to obtain and maintain regulatory approval of
product candidates, the ability to obtain and maintain intellectual
property protection for product candidates and technology, trends
and challenges in the business and markets in which REGENXBIO
operates, the size and growth of potential markets for product
candidates and the ability to serve those markets, the rate and
degree of acceptance of product candidates, and other factors, many
of which are beyond the control of REGENXBIO. Refer to the "Risk
Factors" and "Management's Discussion and Analysis of Financial
Condition and Results of Operations" sections of REGENXBIO's Annual
Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors"
sections of REGENXBIO's Quarterly Reports on Form 10-Q and other
filings, which have been filed with the SEC and are available on
the SEC's website at WWW.SEC.GOV. All of the forward-looking
statements made in this press release are expressly qualified by
the cautionary statements contained or referred to herein. The
actual results or developments anticipated may not be realized or,
even if substantially realized, they may not have the expected
consequences to or effects on REGENXBIO or its businesses or
operations. Such statements are not guarantees of future
performance and actual results or developments may differ
materially from those projected in the forward-looking statements.
Readers are cautioned not to rely too heavily on the
forward-looking statements contained in this press release. These
forward-looking statements speak only as of the date of this press
release. Except as required by law, REGENXBIO does not undertake
any obligation, and specifically declines any obligation, to update
or revise any forward-looking statements, whether as a result of
new information, future events or otherwise.
Zolgensma® is a registered trademark of Novartis Gene
Therapies. All other trademarks referenced herein are registered
trademarks of REGENXBIO.
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
George E. MacDougall
Investor Relations
IR@regenxbio.com
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