Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026
Avidity Biosciences (Nasdaq: RNA) announced a positive pre-BLA meeting with the U.S. FDA for delpacibart zotadirsen (del-zota) in Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). The company updated its planned BLA submission timing to Q1 2026 (previously year-end 2025) to provide additional CMC data requested by FDA. Del-zota holds Breakthrough Therapy designation; Avidity plans this as the first of three BLA submissions over a 12-month period and is preparing a confirmatory study for full approval.
Avidity Biosciences (Nasdaq: RNA) ha annunciato un incontro pre-BLA positivo con la FDA degli Stati Uniti per delpacibart zotadirsen (del-zota) nella distrofia muscolare di Duchenne che offre la possibilità di skip dell’esone 44 (DMD44). L’azienda ha aggiornato i tempi di presentazione della BLA a Q1 2026 (in precedenza fine 2025) per fornire ulteriori dati CMC richiesti dalla FDA. Del-zota detiene la designazione di Terapia Innovativa; Avidity prevede che questa sia la prima di tre presentazioni BLA in un periodo di 12 mesi e sta preparando uno studio di conferma per l’approvazione completa.
Avidity Biosciences (Nasdaq: RNA) anunció una reunión pre-BLA positiva con la FDA de EE. UU. para delpacibart zotadirsen (del-zota) en la distrofia muscular de Duchenne susceptible de omitir el exón 44 (DMD44). La empresa actualizó el plazo previsto para la presentación de la BLA a Q1 2026 (anteriormente finales de 2025) para proporcionar los datos CMC solicitados por la FDA. Del-zota posee la designación de Terapia Innovadora; Avidity lo considera el primero de tres envíos de BLA en un periodo de 12 meses y está preparando un estudio de confirmación para la aprobación completa.
Avidity Biosciences (나스닥: RNA)은 미국 FDA와의 예비 BLA 회의에서 exon 44 건건 생략이 가능한 두수근성 근이영양증(DMD44)에 대한 delpacibart zotadirsen(del-zota)에 대해 긍정적인 결과를 발표했습니다. 회사는 FDA가 요청한 추가 CMC 데이터를 제공하기 위해 Q1 2026를 BLA 제출 시기로 업데이트했습니다(이전에는 2025년 말). Del-zota는 혁신 치료제 지정를 보유하고 있으며, Avidity는 이것을 12개월 동안 세 번의 BLA 제출 중 처음으로 삼고 있으며 완전한 승인을 위한 확인 연구를 준비 중입니다.
Avidity Biosciences (Nasdaq: RNA) a annoncé une réunion pré-BLA positive avec la FDA américaine pour delpacibart zotadirsen (del-zota) dans la dystrophie myopathique de Duchenne éligible à l’exon 44 (DMD44). L’entreprise a mis à jour le calendrier prévu de soumission de la BLA à Q1 2026 (auparavant fin 2025) afin de fournir des données CMC supplémentaires demandées par la FDA. Del-zota bénéficie d’une désignation de thérapie innovante ; Avidity prévoit cela comme la première de trois soumissions BLA sur une période de 12 mois et prépare une étude de confirmation pour une approbation complète.
Avidity Biosciences (Nasdaq: RNA) gab positive Vor-BLA-Besprechung mit der US-amerikanischen FDA für Delpacibart Zotadirsen (del-zota) bei Duchenne-Muskeldystrophie, die durch Exon-44-Sprung behandelbar ist (DMD44). Das Unternehmen aktualisierte den geplanten Zeitpunkt der BLA-Einreichung auf Q1 2026 (zuvor Ende 2025), um zusätzliche CMC-Daten bereitzustellen, die von der FDA angefordert wurden. Del-zota hält die Durchbruch-Therapien-Bezeichnung; Avidity plant dies als die erste von drei BLA-Einreichungen über einen Zeitraum von 12 Monaten und bereitet eine Bestätigungsstudie für die vollständige Zulassung vor.
Avidity Biosciences (Nasdaq: RNA) أعلنت عن اجتماع تمهيدي إيجابي لـ BLA مع إدارة الغذاء والدواء الأمريكية لـ delpacibart zotadirsen (del-zota) في دَوشين مرض ضمور العضلات الدوشيني الذي يمكن علاجه عبر تخطي الإكسون 44 (DMD44). قامت الشركة بتحديث توقيت تقديم BLA المخطط إلى الربع الأول من 2026 (كان سابقاً نهاية 2025) لتوفير بيانات CMC إضافية طلبتها FDA. تُحوز Del-zota تصنيف العلاج الرائد؛ تخطط Avidity لهذا باعتباره الأول من ثلاثة تقديمات BLA خلال فترة 12 شهراً وتجهّز دراسة تأكيدية للحصول على موافقة كاملة.
Avidity Biosciences (纳斯达克：RNA)宣布与美国 FDA 就 delpacibart zotadirsen（del-zota）在杜氏肌营养不良症（DMD44）中通过跳跃外显子44实现治疗的前-BLA 会议取得积极进展。公司将计划中的 BLA 提交时间更新为 2026 年第一季度（原为 2025 年底），以提供 FDA 要求的额外 CMC 数据。Del-zota 拥有 突破性治疗药物指定；Avidity 将其视为在 12 个月内三次 BLA 提交中的第一项，并正在为全面批准准备一项确认性研究。
- Breakthrough Therapy designation for del-zota
- Planned BLA submission in Q1 2026
- First of three planned BLA submissions over 12 months
- FDA meeting described as collaborative with clear path
- BLA timing delayed from year-end 2025 to Q1 2026
- FDA requested additional CMC data at submission
- Confirmatory study required to support full global approval
Insights
Positive FDA pre-BLA alignment; submission timing moved to
The company completed a described "positive" pre-BLA meeting with the U.S. FDA about filing a BLA for del-zota, an investigational therapy for DMD44 that holds Breakthrough Therapy designation. Management reports the FDA gave clear guidance on the additional chemistry, manufacturing, and controls data required at submission, and the firm updated filing timing from year-end 2025 to
Key dependencies and risks are explicit: the FDA requested additional CMC data, and Avidity notes that those materials and other submission elements "may not be satisfactory to the FDA." The company also states it is preparing a confirmatory study to support full global approval and acknowledges reliance on third parties for testing and manufacturing. These facts mean the filing hinge rests on producing CMC and clinical confirmatory evidence acceptable to regulators.
Concrete items to watch: successful delivery of the requested CMC package and the formal BLA filing in
The timing for the BLA submission has been updated to Q1 2026 from previous guidance of year end 2025 to ensure the FDA receives additional data to support the chemistry, manufacturing, and controls (CMC) package at time of submission.
"Our recent meeting with the FDA was highly collaborative and provided a clear path forward for our BLA submission," said Sarah Boyce, President and CEO, Avidity Biosciences. "For DMD, the accelerated approval pathway in the US is the best and fastest way to bring del-zota to people who need it. We are grateful the agency gave us clear guidance on the CMC data it needs at submission, and we believe this alignment can facilitate a successful BLA process. We greatly appreciate the dedicated, diligent reviewers from multiple functions within FDA who are enabling us to move this program forward with the urgency it deserves. We know that every minute matters for boys and young men living with DMD."
Avidity remains highly confident in the potential of del-zota and looks forward to filing a BLA for del-zota in Q1 2026, which will be the Company's first of three planned BLA submissions over a 12-month period. Additionally, Avidity continues to prepare a confirmatory study to support full global approval of del-zota.
About Avidity
Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit
www.aviditybiosciences.com
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Forward-Looking Statements
Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the characterization of the pre-BLA meeting with the FDA; the anticipated timing of a BLA submission for del-zota; the likelihood of approval of a BLA submission for del-zota; the status, progress and potential of del-zota; and Avidity's platform, planned operations and programs. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the additional CMC data to be submitted by Avidity as requested by the FDA, among other data and information to be included in a BLA for del-zota, may not be satisfactory to the FDA; preliminary results of a clinical trial are not necessarily indicative of final results; further analysis of existing clinical data and analysis of new data may lead to conclusions different from those established as of the data cutoff dates in the clinical trial of del-zota, and such data may not meet Avidity's or FDA's expectations; unexpected adverse side effects to, or inadequate efficacy of, del-zota that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA that could be inconsistent with the feedback received to date regarding del-zota and which could delay its currently anticipated timelines; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven; potential delays in the EXPLORE44-OLE™ study; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in
Investor Contact:
Kat Lange
(619) 837-5014
investors@aviditybio.com
Media Contact:
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(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.
FAQ
What did Avidity (RNA) announce about del-zota on October 13, 2025?
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