REGENXBIO Announces FDA Acceptance and Priority Review of the BLA for RGX-121 for MPS II
The FDA has accepted and granted Priority Review for REGENXBIO's (NASDAQ: RGNX) Biologics License Application (BLA) for RGX-121, a potential first-in-class gene therapy for Hunter syndrome (MPS II). The FDA has set a PDUFA target action date of November 9, 2025.
RGX-121 has received multiple FDA designations including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and RMAT. Under a partnership announced in January 2025, NS Pharma (Nippon Shinyaku subsidiary) will lead U.S. commercialization post-approval, while REGENXBIO maintains commercial manufacturing and supply chain responsibilities. If approved, REGENXBIO would retain rights to a potential Priority Review Voucher.
The one-time treatment aims to address both neurodevelopmental and systemic effects of Hunter syndrome, potentially replacing current weekly enzyme replacement therapy.La FDA ha accettato e concesso la Revisione Prioritaria per la Domanda di Licenza Biologica (BLA) di REGENXBIO (NASDAQ: RGNX) relativa a RGX-121, una possibile terapia genica innovativa per la sindrome di Hunter (MPS II). La FDA ha fissato una data obiettivo PDUFA per il 9 novembre 2025.
RGX-121 ha ricevuto diverse designazioni dalla FDA, tra cui Farmaco Orfano, Malattia Pediatrica Rara, Fast Track e RMAT. In base a una collaborazione annunciata a gennaio 2025, NS Pharma (sussidiaria di Nippon Shinyaku) guiderà la commercializzazione negli Stati Uniti dopo l'approvazione, mentre REGENXBIO manterrà le responsabilità relative alla produzione commerciale e alla catena di approvvigionamento. Se approvato, REGENXBIO manterrà i diritti su un potenziale Voucher di Revisione Prioritaria.
Il trattamento, somministrato una sola volta, mira a contrastare sia gli effetti neuroevolutivi sia quelli sistemici della sindrome di Hunter, potenzialmente sostituendo l'attuale terapia sostitutiva enzimatica settimanale.
La FDA ha aceptado y otorgado la Revisión Prioritaria para la Solicitud de Licencia Biológica (BLA) de REGENXBIO (NASDAQ: RGNX) para RGX-121, una posible terapia génica pionera para el síndrome de Hunter (MPS II). La FDA ha establecido una fecha objetivo PDUFA para el 9 de noviembre de 2025.
RGX-121 ha recibido múltiples designaciones de la FDA, incluyendo Medicamento Huérfano, Enfermedad Pediátrica Rara, Vía Rápida y RMAT. Bajo una asociación anunciada en enero de 2025, NS Pharma (subsidiaria de Nippon Shinyaku) liderará la comercialización en EE. UU. tras la aprobación, mientras que REGENXBIO mantendrá las responsabilidades de fabricación comercial y cadena de suministro. Si se aprueba, REGENXBIO conservará los derechos sobre un posible Vale de Revisión Prioritaria.
El tratamiento único busca abordar tanto los efectos neurodesarrollativos como sistémicos del síndrome de Hunter, potencialmente reemplazando la terapia de reemplazo enzimático semanal actual.
FDA가 REGENXBIO(NASDAQ: RGNX)의 생물학적 제제 허가 신청서(BLA)인 RGX-121에 대해 우선 심사(Priority Review)를 승인했습니다. RGX-121은 헌터 증후군(MPS II)에 대한 최초의 혁신적 유전자 치료제로 기대됩니다. FDA는 2025년 11월 9일을 PDUFA 목표 심사 완료일로 지정했습니다.
RGX-121은 희귀의약품(Orphan Drug), 희귀 소아 질환(Rare Pediatric Disease), 신속 심사(Fast Track), RMAT 등 여러 FDA 지정 혜택을 받았습니다. 2025년 1월 발표된 파트너십에 따라 NS Pharma(니폰 시냐쿠 자회사)가 승인 후 미국 내 상업화를 주도하며, REGENXBIO는 상업 생산과 공급망 관리를 담당합니다. 승인 시 REGENXBIO는 우선 심사 바우처 권리를 유지합니다.
이 1회 치료는 헌터 증후군의 신경발달 및 전신적 영향을 모두 개선하는 것을 목표로 하며, 현재 매주 시행되는 효소 대체 치료를 대체할 가능성이 있습니다.
La FDA a accepté et accordé une Revue Prioritaire pour la Demande d'Autorisation de Mise sur le Marché Biologique (BLA) de REGENXBIO (NASDAQ : RGNX) concernant RGX-121, une thérapie génique potentiellement innovante pour le syndrome de Hunter (MPS II). La FDA a fixé une date cible PDUFA au 9 novembre 2025.
RGX-121 a reçu plusieurs désignations de la FDA, notamment Médicament Orphelin, Maladie Pédiatrique Rare, Fast Track et RMAT. Dans le cadre d'un partenariat annoncé en janvier 2025, NS Pharma (filiale de Nippon Shinyaku) prendra en charge la commercialisation aux États-Unis après approbation, tandis que REGENXBIO conservera les responsabilités liées à la fabrication commerciale et à la chaîne d'approvisionnement. En cas d'approbation, REGENXBIO conservera les droits sur un éventuel Voucher de Revue Prioritaire.
Ce traitement unique vise à traiter à la fois les effets neurodéveloppementaux et systémiques du syndrome de Hunter, pouvant potentiellement remplacer la thérapie enzymatique hebdomadaire actuelle.
Die FDA hat die Zulassungsanmeldung (BLA) von REGENXBIO (NASDAQ: RGNX) für RGX-121, eine potenzielle bahnbrechende Gentherapie für das Hunter-Syndrom (MPS II), zur Prioritätsprüfung akzeptiert und genehmigt. Die FDA hat ein PDUFA-Zieldatum für den 9. November 2025 festgelegt.
RGX-121 erhielt mehrere FDA-Designationen, darunter Orphan Drug, Rare Pediatric Disease, Fast Track und RMAT. Im Rahmen einer im Januar 2025 angekündigten Partnerschaft wird NS Pharma (Tochtergesellschaft von Nippon Shinyaku) die Vermarktung in den USA nach der Zulassung übernehmen, während REGENXBIO die kommerzielle Herstellung und Lieferkette verantwortet. Bei Zulassung behält REGENXBIO die Rechte an einem potenziellen Priority Review Voucher.
Die einmalige Behandlung zielt darauf ab, sowohl neuroentwicklungsbedingte als auch systemische Auswirkungen des Hunter-Syndroms zu adressieren und könnte die derzeitige wöchentliche Enzymersatztherapie ersetzen.
- FDA granted Priority Review status, accelerating the review process
- RGX-121 could be the first gene therapy for MPS II (Hunter syndrome)
- Multiple FDA designations received (Orphan Drug, Rare Pediatric Disease, Fast Track, RMAT)
- Strategic partnership with Nippon Shinyaku for commercialization secured
- Potential to receive valuable Priority Review Voucher upon approval
- None.
Insights
FDA's Priority Review for REGENXBIO's MPS II gene therapy accelerates potential approval, positioning the company for a first-to-market breakthrough treatment.
The FDA's acceptance and Priority Review of REGENXBIO's Biologics License Application (BLA) for RGX-121 represents a significant regulatory milestone that substantially accelerates the review timeline. With a PDUFA date of November 9, 2025, the company has a clearly defined regulatory pathway that typically shortens the standard 10-month review to just 6 months, reflecting the FDA's recognition of the therapy's potential importance.
This announcement carries particular significance as RGX-121 aims to become the first gene therapy treatment for MPS II (Hunter syndrome), a rare lysosomal storage disorder. Current standard of care requires burdensome weekly enzyme replacement therapy that doesn't adequately address neurodevelopmental manifestations of the disease. RGX-121's one-time treatment approach could represent a paradigm shift in treatment modality.
The therapy's impressive regulatory designation portfolio – Orphan Drug, Rare Pediatric Disease, Fast Track, and RMAT designations from the FDA, plus ATMP classification from the EMA – demonstrates the regulatory bodies' acknowledgment of both the unmet medical need and the promising clinical data.
The commercial partnership with Nippon Shinyaku establishes a clear commercialization strategy, with NS Pharma handling US commercialization while REGENXBIO manages manufacturing and supply chain. Additionally, approval could yield a Priority Review Voucher, which REGENXBIO retains full rights to sell – these vouchers have historically sold for
- 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
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
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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SOURCE REGENXBIO Inc.
FAQ
When is the PDUFA date for REGENXBIO's RGX-121 gene therapy for Hunter syndrome?
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