Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1
Dyne Therapeutics (Nasdaq: DYN) has received FDA Fast Track designation for DYNE-101, their treatment candidate for myotonic dystrophy type 1 (DM1). The designation follows positive clinical data from their ongoing Phase 1/2 ACHIEVE global trial, which showed substantial functional benefits and splicing correction in patients.
The company plans to submit for U.S. Accelerated Approval in H1 2026. The Fast Track status enables more frequent FDA communications, rolling review of marketing applications, and potential eligibility for Accelerated Approval and Priority Review. This development pathway aims to expedite the delivery of DYNE-101 to DM1 patients, for whom there are currently no approved therapies.
Dyne Therapeutics (Nasdaq: DYN) ha ricevuto la designazione di Fast Track dalla FDA per DYNE-101, il loro candidato terapeutico per la distrofia miotonica di tipo 1 (DM1). Questa designazione segue dati clinici positivi del loro trial globale di Fase 1/2 denominato ACHIEVE, che ha mostrato sostanziali benefici funzionali e una correzione di splicing nei pazienti.
L'azienda prevede di presentare la domanda per l'Approvazione Accelerata negli Stati Uniti nel H1 2026. Lo stato di Fast Track consente comunicazioni più frequenti con la FDA, una revisione continua delle applicazioni di marketing e la potenziale idoneità per l'Approvazione Accelerata e la Revisione Prioritaria. Questo percorso di sviluppo mira a velocizzare la consegna di DYNE-101 ai pazienti affetti da DM1, per i quali attualmente non esistono terapie approvate.
Dyne Therapeutics (Nasdaq: DYN) ha recibido la designación de Fast Track de la FDA para DYNE-101, su candidato de tratamiento para la distrofia miotónica tipo 1 (DM1). Esta designación sigue a datos clínicos positivos de su ensayo global en Fase 1/2 denominado ACHIEVE, que mostró beneficios funcionales sustanciales y corrección de splicing en los pacientes.
La compañía planea presentar la solicitud para la Aprobación Acelerada en EE. UU. en H1 2026. El estatus de Fast Track permite comunicaciones más frecuentes con la FDA, revisión continua de las aplicaciones de comercialización y potencial elegibilidad para la Aprobación Acelerada y la Revisión Prioritaria. Este camino de desarrollo tiene como objetivo acelerar la entrega de DYNE-101 a los pacientes con DM1, para quienes actualmente no existen terapias aprobadas.
Dyne Therapeutics (Nasdaq: DYN)는 DYNE-101에 대해 FDA의 패스트 트랙 지정을 받았습니다. 이는 그들의 치료 후보 물질로서, 1형 미오토닉 근육병(DM1)을 치료하기 위한 것입니다. 이 지명은 진행 중인 글로벌 1/2상 ACHIEVE 임상 시험에서 긍정적인 결과를 바탕으로 이루어졌으며, 환자들에게 상당한 기능적 이점과 스플라이싱 교정을 보여주었습니다.
회사는 2026년 1분기(H1 2026)에 미국에서의 가속 승인을 신청할 계획입니다. 패스트 트랙 상태는 FDA와의 더 빈번한 소통, 마케팅 신청서의 계속적인 검토 및 가속 승인과 우선 심사 자격 가능성을 제공합니다. 이 개발 경로는 DM1 환자들에게 DYNE-101의 공급을 신속하게 진행하는 것을 목표로 하고 있으며, 현재 승인을 받은 치료법이 없습니다.
Dyne Therapeutics (Nasdaq: DYN) a reçu la désignation Fast Track de la FDA pour DYNE-101, leur candidat traitement pour la dystrophie myotonique de type 1 (DM1). Cette désignation fait suite à des données cliniques positives de leur essai mondial de Phase 1/2 nommé ACHIEVE, qui a montré des bénéfices fonctionnels substantiels et une correction de splicing chez les patients.
L'entreprise prévoit de soumettre une demande d'Approbation Accélérée aux États-Unis au H1 2026. Le statut Fast Track permet des communications plus fréquentes avec la FDA, un examen continu des demandes de marketing et une éventuelle éligibilité à l'Approbation Accélérée et à l'Examen Prioritaire. Ce parcours de développement vise à accélérer la livraison de DYNE-101 aux patients atteints de DM1, pour lesquels il n'existe actuellement aucune thérapie approuvée.
Dyne Therapeutics (Nasdaq: DYN) hat von der FDA den Fast Track-Status für DYNE-101 erhalten, ihren Behandlungskandidaten für die myotonische Dystrophie Typ 1 (DM1). Diese Auszeichnung folgt auf positive klinische Daten aus ihrer laufenden globalen Phase 1/2 ACHIEVE-Studie, die erhebliche funktionale Vorteile und eine Splicing-Korrektur bei den Patienten zeigten.
Das Unternehmen plant, im H1 2026 einen Antrag auf beschleunigte Zulassung in den USA einzureichen. Der Fast Track-Status ermöglicht häufigere Kommunikation mit der FDA, eine fortlaufende Überprüfung von Marketinganträgen und potenzielle Berechtigung für beschleunigte Zulassung und priorisierte Überprüfung. Dieser Entwicklungsweg zielt darauf ab, die Bereitstellung von DYNE-101 für DM1-Patienten zu beschleunigen, für die derzeit keine zugelassenen Therapien verfügbar sind.
- Received FDA Fast Track designation for DYNE-101
- Demonstrated substantial functional benefits in ACHIEVE trial
- Showed positive splicing correction biomarker results
- Eligible for accelerated approval pathway
- Rolling review possibility for marketing application
- None.
Insights
The Fast Track designation for DYNE-101 represents a significant regulatory milestone that could accelerate the path to market. The FDA's decision appears driven by robust clinical data from the ACHIEVE trial, which demonstrated both functional benefits and biomarker improvements in DM1 patients.
The accelerated timeline targeting a BLA submission in H1 2026 is particularly noteworthy. The rolling review benefit could potentially reduce the traditional review period by several months. For context, DM1 affects approximately 1 in 8,000 people globally, representing a substantial market opportunity in a disease area with no approved treatments.
In simple terms: Think of Fast Track as a 'priority lane' at the airport security check. Instead of waiting in the regular line (standard FDA review), Dyne can move through regulatory checkpoints more quickly and submit their application in parts rather than all at once.
The clinical implications of this development are substantial. Myotonic Dystrophy Type 1 (DM1) is a progressive muscle-wasting disorder that severely impacts patient quality of life. DYNE-101's mechanism targeting splicing correction addresses the fundamental molecular defect in DM1.
The mention of 'substantial functional benefit' and 'compelling effect on splicing correction' in the ACHIEVE trial suggests meaningful therapeutic efficacy. These outcomes are particularly important as DM1 patients currently rely solely on symptomatic management.
For the average person: Imagine your muscles are like a malfunctioning light switch that can't turn off properly - that's what happens in DM1. DYNE-101 works to fix the switch itself, not just deal with the consequences of it being stuck.
This regulatory advancement significantly de-risks Dyne's development pathway and could accelerate market entry by months or even years. With a market cap of
The orphan disease market typically commands premium pricing, often exceeding
For everyday investors: This is like getting a green light to use the express lane on a highway, potentially getting the drug to market faster and starting to generate revenue sooner than expected.
- Company Anticipates Submitting for U.S. Accelerated Approval in H1 2026 -
WALTHAM, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is currently being evaluated in the ongoing Phase 1/2 ACHIEVE global clinical trial.
“This Fast Track designation comes on the heels of robust clinical data from our ACHIEVE trial, which demonstrated substantial functional benefit for patients across a range of clinical measures and a compelling effect on the key disease biomarker of splicing correction,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “DM1 is a devastating disease with no approved therapies, and we are driven to deliver DYNE-101, a potentially transformative medicine, to patients as quickly as possible.”
The FDA grants Fast Track designation to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. Rolling review allows a company to submit completed sections of its Biologic License Application (BLA) for review by FDA as they are ready, rather than waiting until the entire application is complete for FDA review. This may lead to earlier drug approval and access by patients. A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met.
About DYNE-101
DYNE-101 is an investigational therapeutic being evaluated in the Phase 1/2 global ACHIEVE clinical trial for people living with DM1. DYNE-101 consists of an antisense oligonucleotide (ASO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease progression. DYNE-101 has been granted orphan drug designation by the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of DM1.
About Myotonic Dystrophy Type 1 (DM1)
DM1 is a rare, progressive, genetic disease that affects skeletal, cardiac and smooth muscle. It is a monogenic, autosomal dominant disease caused by an abnormal trinucleotide expansion in a region of the DMPK gene. This expansion of CTG repeats causes toxic RNA to cluster in the nucleus, forming nuclear foci and altering the splicing of multiple proteins essential for normal cellular function. This altered splicing, or spliceopathy, results in a wide range of symptoms. People living with DM1 typically experience myotonia and progressive weakness of major muscle groups, which can affect mobility, breathing, heart function, speech, digestion and vision as well as cognition. DM1 is estimated to affect more than 40,000 people in the United States and over 74,000 people in Europe, but there are currently no approved disease-modifying therapies.
About Dyne Therapeutics
Dyne Therapeutics is focused on discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that are designed to overcome limitations in delivery to muscle tissue and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. For more information, please visit 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 anticipated timeline for reporting data from the DYNE-101 ACHIEVE clinical trial and expectations regarding the potential benefits of fast track designation, and expectations regarding the timing and outcome of interactions with global regulatory authorities and the availability of accelerated approval pathways for DYNE-101 and expectations regarding the timing of filing applications for U.S. Accelerated Approval 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,” 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 contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne’s ability to enroll patients in clinical trials; whether results from preclinical studies and initial data from early clinical trials will be predictive of the final results of the clinical trials or future trials; uncertainties as to the FDA’s and other regulatory authorities’ interpretation of the data from Dyne’s clinical trials and acceptance of Dyne’s clinical programs and the regulatory approval process; fast track designation by the FDA may not actually lead to a faster development or regulatory review or approval process; whether Dyne’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne’s filings with the Securities and Exchange Commission (SEC), including Dyne’s most recent Form 10-Q and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne’s views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne’s views as of any date subsequent to the date of this press release.
Contacts:
Investors
Mike Hencke
Kendall Investor Relations
ir@dyne-tx.com 
Media
Stacy Nartker
Dyne Therapeutics
snartker@dyne-tx.com
781-317-1938
FAQ
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