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Dyne的DMD候选药z-rostudirsen获FDA受理BLA并授予优先审评
这是Dyne Therapeutics（DYN.US）z-rostudirsen从提交BLA推进到FDA正式受理并给出2027-01-21 PDUFA的正向监管节点。
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不做 Dyne Therapeutics（DYN.US）
不做：FDA受理和Priority Review方向正面但属5月BLA提交后预期内程序性落地，批准标准和2027年PDUFA风险仍在；盘前已涨约4.5%、20日涨约23.7%，开盘追高的剩余edge不足。
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研究档案
消息传播链
- 公司已披露z-rostudirsen BLA提交、申请Priority Review，并预期若按计划获批可在2027年一季度上市。
- 公司宣布FDA受理BLA、授予Priority Review，并设定PDUFA目标日为2027-01-21。市场反应:DYN.US最新价23.74美元，较前收22.72美元上涨4.49%。
- FDA说明Priority Review目标是在6个月内行动，但不改变批准所需科学和医学标准。
关键理由
- 这是Dyne Therapeutics（DYN.US）z-rostudirsen从提交BLA推进到FDA正式受理并给出2027-01-21 PDUFA的正向监管节点。
- 但公司在2026-05-26提交BLA时已明确申请Priority Review并预期若获批可在2027年一季度上市，本次更多是预期内程序性落地。
- 量级上，DMD美国约1.2万人、exon 51约占一成多，Dyne此前称约1600人的既有市场；若未来获批才可能形成数亿美元级年销售机会，相比当前约39亿美元市值有意义，但今天不是批准。
主要风险
- FDA优先审评只缩短审评目标时间，不降低批准标准， accelerated approval 仍有surrogate endpoint和临床获益争议。
- DYN.US盘前已按该消息上涨约4.5%、20日上涨约23.7%且接近52周高位，开盘追程序性节点容易变成高开低走。
反向证据
- 做多前查反向 · 来源 web · 日期 2026-06 · 影响 weakens
DMD exon-skipping同类疗法依赖dystrophin替代终点，历史上存在FDA审评争议；本次受理不等于批准。
检索:Dyne Therapeutics z-rostudirsen risks FDA approval exon 51 DMD analysts July 2026 原文 ↗ - 做多前查反向 · 来源 kb_search · 影响 none
内部KB未命中针对本次受理节点或商业化增量的强新增付费研究证据。
检索:Dyne Therapeutics z-rostudirsen BLA accepted Priority Review DMD exon 51 market sales competition
工具调用 · 历史记录,结果未知(4 种)
- search_processed_events · 结果未知
- stock_quote · 结果未知
- kb_search · 结果未知
- search_case_library · 结果未知
原文全文
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Poised to address unmet need in neuromuscular diseases and
positioned for growth
Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
July 20, 2026
PDF Version
- Priority Review granted; PDUFA target action date set for January 21, 2027 -
- Submission for Accelerated Approval based on dystrophin as a surrogate endpoint -
- In the registrational expansion cohort of the DELIVER trial, treatment with z-rostudirsen once every 4 weeks resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile 1 -
WALTHAM, Mass., July 20, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc . (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The FDA has granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 21, 2027. Dyne continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming approval is received on the anticipated timeline.
“This milestone represents significant progress toward our goal of delivering functional improvement for those living with DMD amenable to exon 51 skipping,” said John Cox, president and chief executive officer of Dyne. “With z-rostudirsen, we set out to advance the treatment paradigm in DMD by combining a robust increase in near-full length dystrophin with broad delivery to relevant tissues. With a potential approval in six months, we are continuing our launch preparations with the aim of making z-rostudirsen available as quickly as possible. We are deeply grateful to the entire Duchenne community, whose partnership and support have been essential in developing this potential therapy.”
In addition to z-rostudirsen, Dyne is advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the potential treatment of DMD amenable to skipping of exons 53, 45, 44, and 55, respectively.
About Zeleciment Rostudirsen (z-rostudirsen, also known as DYNE-251)
Z-rostudirsen is an investigational therapeutic for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. The registrational expansion cohort of the global Phase 1/2 DELIVER clinical trial of z-rostudirsen met its primary endpoint. Data from the DELIVER trial served as the basis for a Biologics License Application (BLA) for potential U.S. Accelerated Approval, which has been granted Priority Review. The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Z-rostudirsen continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial.
Z-rostudirsen consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). It is designed to enable the production of near-full length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement. Z-rostudirsen has received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the FDA, European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan for the treatment of individuals with DMD amenable to exon 51 skipping.
In addition to z-rostudirsen, Dyne is building a DMD franchise and has preclinical programs targeting other exons, including DYNE-253, DYNE-245, DYNE-244 and DYNE-255.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. These mutations result in a complete or near-complete absence of dystrophin, a protein critical for maintaining muscle structure and function. DMD is the most common form of childhood-onset muscular dystrophy, affecting approximately 12,000 individuals in the U.S. and 16,000 in the EU. Symptoms typically emerge between ages 3 and 5 and include progressive muscle weakness, loss of lower and upper limb function and eventually cardiac and respiratory failure. In addition to physical decline, individuals may experience cognitive impairment and neuropsychiatric challenges such as intellectual disabilities, learning difficulties and behavioral disorders. Despite existing therapies, there remains a significant unmet need for new treatment options that deliver functional improvement.
About Dyne Therapeutics
Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease. The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more at https://www.dyne-tx.com/ , and follow us on X , LinkedIn and Facebook .
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the clinical and therapeutic potential of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), the potential of z-rostudirsen to advance the treatment paradigm in Duchenne muscular dystrophy, expectations regarding the timing and outcome of interactions with regulatory authorities and the timing of regulatory authority action, and expectations regarding the timing of commercialization of z-rostudirsen, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” “will” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements co
Skip to main content
Open navigation
About Us
FORCE TM Platform
Pipeline
Physicians
Patients
Investors & Media
Culture & Careers
Overview
Leadership
Board of directors
Scientific advisory board
Corporate Responsibility
Contact us
about-us
Overview
Scientific publications & presentations
force™-platform
Overview
Zeleciment rostudirsen (DYNE-251)
Zeleciment basivarsen (DYNE-101)
DYNE-302
pipeline
physicians
Commitment
Clinical trials
DMD stories
DM1 stories
FSHD stories
Resources
patients
Company overview
News, publications & events
Stock information
Corporate governance
Financials & filings
IR resources
investors--media
Our culture & values
Hear from our Dynamos
Competitive benefits
Open positions
culture--careers
Stay in Touch
info@dyne-tx.com
781.786.8230
781.786.8230
linkedin.com
twitter.com
facebook.com
youtube.com
Poised to address unmet need in neuromuscular diseases and
positioned for growth
Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
July 20, 2026
PDF Version
- Priority Review granted; PDUFA target action date set for January 21, 2027 -
- Submission for Accelerated Approval based on dystrophin as a surrogate endpoint -
- In the registrational expansion cohort of the DELIVER trial, treatment with z-rostudirsen once every 4 weeks resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile 1 -
WALTHAM, Mass., July 20, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc . (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The FDA has granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 21, 2027. Dyne continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming approval is received on the anticipated timeline.
“This milestone represents significant progress toward our goal of delivering functional improvement for those living with DMD amenable to exon 51 skipping,” said John Cox, president and chief executive officer of Dyne. “With z-rostudirsen, we set out to advance the treatment paradigm in DMD by combining a robust increase in near-full length dystrophin with broad delivery to relevant tissues. With a potential approval in six months, we are continuing our launch preparations with the aim of making z-rostudirsen available as quickly as possible. We are deeply grateful to the entire Duchenne community, whose partnership and support have been essential in developing this potential therapy.”
In addition to z-rostudirsen, Dyne is advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the potential treatment of DMD amenable to skipping of exons 53, 45, 44, and 55, respectively.
About Zeleciment Rostudirsen (z-rostudirsen, also known as DYNE-251)
Z-rostudirsen is an investigational therapeutic for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. The registrational expansion cohort of the global Phase 1/2 DELIVER clinical trial of z-rostudirsen met its primary endpoint. Data from the DELIVER trial served as the basis for a Biologics License Application (BLA) for potential U.S. Accelerated Approval, which has been granted Priority Review. The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Z-rostudirsen continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial.
Z-rostudirsen consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). It is designed to enable the production of near-full length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement. Z-rostudirsen has received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the FDA, European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan for the treatment of individuals with DMD amenable to exon 51 skipping.
In addition to z-rostudirsen, Dyne is building a DMD franchise and has preclinical programs targeting other exons, including DYNE-253, DYNE-245, DYNE-244 and DYNE-255.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. These mutations result in a complete or near-complete absence of dystrophin, a protein critical for maintaining muscle structure and function. DMD is the most common form of childhood-onset muscular dystrophy, affecting approximately 12,000 individuals in the U.S. and 16,000 in the EU. Symptoms typically emerge between ages 3 and 5 and include progressive muscle weakness, loss of lower and upper limb function and eventually cardiac and respiratory failure. In addition to physical decline, individuals may experience cognitive impairment and neuropsychiatric challenges such as intellectual disabilities, learning difficulties and behavioral disorders. Despite existing therapies, there remains a significant unmet need for new treatment options that deliver functional improvement.
About Dyne Therapeutics
Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. We are developing therapeutics that target muscle and the central nervous system (CNS) to address the root cause of disease. The company is advancing clinical programs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) as well as preclinical programs for facioscapulohumeral muscular dystrophy (FSHD), Pompe disease and multiple DMD mutations. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more at https://www.dyne-tx.com/ , and follow us on X , LinkedIn and Facebook .
Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne’s strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform, the clinical and therapeutic potential of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), the potential of z-rostudirsen to advance the treatment paradigm in Duchenne muscular dystrophy, expectations regarding the timing and outcome of interactions with regulatory authorities and the timing of regulatory authority action, and expectations regarding the timing of commercialization of z-rostudirsen, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” “will” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements co
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