Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD)
Dyne Therapeutics (NASDAQ:DYN) has received FDA Breakthrough Therapy Designation for DYNE-251, targeting Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER trial, where DYNE-251 demonstrated sustained functional improvement through 18 months in key measures like time to rise and stride velocity.
The company has completed enrollment of 32 patients in the DELIVER registrational expansion cohort, with data expected in late 2025. Dyne plans to submit a Biologics License Application (BLA) for U.S. accelerated approval in early 2026. DYNE-251 has also received Fast Track, Orphan Drug, and Rare Pediatric disease designations from the FDA.
Dyne Therapeutics (NASDAQ:DYN) ha ottenuto la designazione di terapia innovativa (Breakthrough Therapy Designation) dalla FDA per DYNE-251, destinato ai pazienti con distrofia muscolare di Duchenne (DMD) suscettibili al salto dell'esone 51. Questa designazione si basa sui dati del trial in corso DELIVER, in cui DYNE-251 ha mostrato un miglioramento funzionale sostenuto per 18 mesi in parametri chiave come il tempo per alzarsi e la velocit脿 del passo.
L'azienda ha completato l'arruolamento di 32 pazienti nella coorte di espansione registrazionale DELIVER, con dati attesi per la fine del 2025. Dyne prevede di presentare una domanda di autorizzazione biologica (BLA) per l'approvazione accelerata negli Stati Uniti nei primi mesi del 2026. DYNE-251 ha inoltre ricevuto le designazioni Fast Track, Orphan Drug e Rare Pediatric Disease dalla FDA.
Dyne Therapeutics (NASDAQ:DYN) ha recibido la designaci贸n de Terapia Innovadora (Breakthrough Therapy Designation) por parte de la FDA para DYNE-251, dirigido a pacientes con distrofia muscular de Duchenne (DMD) aptos para el salto del ex贸n 51. La designaci贸n se basa en datos del ensayo en curso DELIVER, donde DYNE-251 demostr贸 una mejora funcional sostenida durante 18 meses en medidas clave como el tiempo para levantarse y la velocidad de zancada.
La compa帽铆a ha completado la inscripci贸n de 32 pacientes en la cohorte de expansi贸n registracional DELIVER, con datos esperados para finales de 2025. Dyne planea presentar una Solicitud de Licencia Biol贸gica (BLA) para la aprobaci贸n acelerada en EE. UU. a principios de 2026. DYNE-251 tambi茅n ha recibido las designaciones Fast Track, Orphan Drug y Enfermedad Pedi谩trica Rara por parte de la FDA.
Dyne Therapeutics (NASDAQ:DYN)臧� FDA搿滊秬韯� 順侅嫚 旃橂鞝� 歆�鞝�(Breakthrough Therapy Designation)鞚� 氚涭晿鞀惦媹雼�. 鞚措姅 鞐戩啇 51 鞀ろ偆頃戩澊 臧�電ロ暅 霋れ叴 攴检澊鞓侅枒歃�(DMD) 頇橃瀽毳� 雽�靸侅溂搿� 頃橂姅 DYNE-251鞐� 雽�頃� 瓴冹瀰雼堧嫟. 鞚� 歆�鞝曥潃 歆勴枆 欷戩澑 DELIVER 鞛勳儊鞁滍棙鞚� 雿办澊韯半ゼ 旮半皹鞙茧 頃橂┌, DYNE-251鞚� 18臧滌洈 霃欖晥 欤检殧 旮半姤 歆�響滌澑 鞚检柎雮橁赴 鞁滉皠瓿� 氤错彮 靻嶋弰鞐愳劀 歆�靻嶌爜鞚� 旮半姤 臧滌劆鞚� 氤挫榾鞚岇潉 雮橅儉雰呺媹雼�.
須岇偓電� DELIVER 霌彪 頇曥灔 旖旐樃韸胳棎靹� 32氇呾潣 頇橃瀽 霌彪鞚� 鞕勲頄堨溂氅�, 雿办澊韯半姅 2025雲� 毵�鞐� 鞓堨儊霅╇媹雼�. Dyne鞚� 2026雲� 齑堨棎 氙戈淡鞐愳劀 臧�靻� 鞀轨澑鞖� 靸濍鞚橃暯頀� 項堦皜 鞁犾箔靹�(BLA)毳� 鞝滌稖頃� 瓿勴殟鞛呺媹雼�. DYNE-251鞚� 霕愴暅 FDA搿滊秬韯� Fast Track, 頋穩 鞚橃暯頀�(Orphan Drug), 頋穩 靻岇晞 歆堩櫂 歆�鞝曥潉 氚涭晿鞀惦媹雼�.
Dyne Therapeutics (NASDAQ:DYN) a re莽u la d茅signation de th茅rapie r茅volutionnaire (Breakthrough Therapy Designation) de la FDA pour DYNE-251, destin茅 aux patients atteints de dystrophie musculaire de Duchenne (DMD) 茅ligibles au saut de l'exon 51. Cette d茅signation repose sur les donn茅es de l'essai en cours DELIVER, o霉 DYNE-251 a d茅montr茅 une am茅lioration fonctionnelle soutenue pendant 18 mois sur des crit猫res cl茅s tels que le temps pour se lever et la vitesse de foul茅e.
La soci茅t茅 a termin茅 l'inclusion de 32 patients dans la cohorte d'expansion d'enregistrement DELIVER, avec des donn茅es attendues pour fin 2025. Dyne pr茅voit de soumettre une demande d'autorisation de mise sur le march茅 biologique (BLA) pour une approbation acc茅l茅r茅e aux 脡tats-Unis au d茅but de 2026. DYNE-251 a 茅galement re莽u les d茅signations Fast Track, Orphan Drug et Maladie p茅diatrique rare de la FDA.
Dyne Therapeutics (NASDAQ:DYN) hat von der FDA die Breakthrough Therapy Designation f眉r DYNE-251 erhalten, das sich an Patienten mit Duchenne-Muskeldystrophie (DMD) richtet, die f眉r ein Exon-51-Skipping geeignet sind. Die Designation basiert auf Daten der laufenden DELIVER-Studie, in der DYNE-251 eine anhaltende funktionelle Verbesserung 眉ber 18 Monate bei wichtigen Messgr枚脽en wie Aufstehzeit und Schritttempo zeigte.
Das Unternehmen hat die Einschreibung von 32 Patienten in der registratorischen Erweiterungskohorte DELIVER abgeschlossen, mit Daten, die f眉r Ende 2025 erwartet werden. Dyne plant, Anfang 2026 einen Biologics License Application (BLA) f眉r eine beschleunigte Zulassung in den USA einzureichen. DYNE-251 hat zudem die FDA-Designationen Fast Track, Orphan Drug und Rare Pediatric Disease erhalten.
- FDA Breakthrough Therapy Designation received for DYNE-251, potentially expediting development and review process
- Sustained functional improvement demonstrated through 18 months in clinical trials
- Multiple regulatory designations secured (Fast Track, Orphan Drug, Rare Pediatric Disease)
- Potential reduction in BLA review timeline from 12 to 8 months
- Enhanced FDA support and guidance for development process
- None.
Insights
FDA Breakthrough Therapy Designation for DYNE-251 significantly accelerates potential approval pathway for Dyne's DMD treatment showing impressive clinical results.
The FDA's Breakthrough Therapy Designation for DYNE-251 represents a significant regulatory milestone for Dyne Therapeutics' Duchenne muscular dystrophy (DMD) program. This designation is not awarded lightly - it requires preliminary clinical evidence indicating substantial improvement over existing therapies on clinically significant endpoints. The fact that DYNE-251 received this designation speaks volumes about the strength of the clinical data Dyne has generated thus far.
The company has disclosed that DYNE-251 demonstrated sustained functional improvement through 18 months across key mobility measures like time to rise and stride velocity. Perhaps more importantly, the drug appears to be producing near-full-length dystrophin - the critical protein missing in DMD patients. This is particularly noteworthy as current approved exon-skipping therapies have struggled with producing sufficient dystrophin levels to deliver meaningful functional benefits.
This designation provides Dyne with tangible regulatory advantages: enhanced FDA support with senior-level involvement, expedited review timelines (potentially reducing BLA review from 12 to 8 months), and eligibility for rolling submission. With enrollment completed for the 32-patient Registrational Expansion Cohort of the DELIVER trial and data expected in late 2025, Dyne appears positioned for a potential BLA submission for accelerated approval by early 2026.
The fact that both of Dyne's lead programs now have Breakthrough Therapy Designation (including DYNE-101 for myotonic dystrophy type 1) suggests their FORCE鈩� platform may be delivering on its promise across multiple neuromuscular indications. For patients with DMD amenable to exon 51 skipping (approximately 13% of DMD patients), DYNE-251 could potentially offer a more effective treatment option than currently available therapies.
- Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 - 聽聽
WALTHAM, Mass., Aug. 04, 2025 (GLOBE NEWSWIRE) -- 听(狈补蝉诲补辩: 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 granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial.
鈥淭his Breakthrough Therapy Designation for DYNE-251 is a testament to its potential as a next-generation therapy designed to bring meaningful functional improvement to individuals with DMD for whom exon 51 skipping can lead to the production of near full-length dystrophin,鈥� said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. 鈥淎s we鈥檝e previously disclosed, DYNE-251 has demonstrated sustained functional improvement through eighteen months, as assessed by key measures such as time to rise and stride velocity 95th centile. The level of near-full-length dystrophin expression observed marks a significant step forward to potentially deliver meaningful benefits to patients.鈥�
Both of Dyne鈥檚 lead programs now have FDA Breakthrough Therapy Designation, with DYNE-101 having been granted Breakthrough Therapy Designation in myotonic dystrophy type 1 (DM1) earlier this year. The FDA grants Breakthrough Therapy Designation to expedite the development and review of drugs that are intended to treat a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy on one or more clinically significant endpoints. This designation offers benefits to DYNE-251 in the U.S. including:聽
- Enhanced FDA support, including senior-level involvement to guide efficient development as well as decision-making聽
- Early and frequent communication with FDA reviewers on trial design and regulatory strategy聽
- Rolling and Priority Review eligibility, potentially reducing the Biologics License Application (BLA) review timeline from 12 to 8 months聽
DYNE-251 in DMD
- DYNE-251 has been granted Fast Track, Orphan Drug and Rare Pediatric disease designations by the FDA and Orphan Drug designation by the European Medicines Agency (EMA) for the treatment of DMD.
- Dyne has completed enrollment of 32 patients in the Registrational Expansion Cohort of the DELIVER trial. Data from this cohort are planned for late 2025.
- Dyne anticipates a potential BLA submission for U.S. Accelerated Approval in early 2026.
- Dyne also continues to pursue approval pathways outside of the U.S. for DYNE-251 in patients with DMD with a confirmed mutation amenable to exon 51 skipping.
About the DELIVER Trial
DELIVER is a global, randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy (as measured by both biomarker and functional improvement) of DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg of DYNE-251 administered every four weeks. A registrational expansion cohort to support a potential regulatory submission for U.S. Accelerated Approval, is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot at 6 months. For more information on the DELIVER trial, visit聽聽(NCT05524883) 补苍诲听听(2023-510351-31-00).
About DYNE-251
DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 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. DYNE-251 has received Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug designation from the FDA and European Medicines Agency (EMA) for the treatment of individuals with DMD.
In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.鈥�
About Duchenne Muscular Dystrophy (DMD)
DMD is a rare, X-linked, recessive, progressive neuromuscular disorder and results from mutations in the DMD gene which lead to total absence or nearly undetectable levels of dystrophin protein, essential for muscle structure, function and preservation. DMD occurs primarily in males and affects approximately 12,000 individuals in the U.S. and approximately 16,000 in the EU. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is 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 myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. At Dyne, we are on a mission to deliver functional improvement for individuals, families and communities. Learn more, and follow us on聽,补苍诲听.听听
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鈥檚 strategy, future operations, prospects and plans, objectives of management; the potential of DYNE-251; the anticipated timelines for reporting additional data from the DELIVER clinical trial, submitting applications for marketing approval and commercial launches; the availability of expedited approval pathways for DYNE-251; and expectations regarding the timing and outcome of interactions with regulatory authorities, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥渕ight,鈥� 鈥渙bjective,鈥� 鈥渙ngoing,鈥� 鈥減lan,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥減otential,鈥� 鈥渟hould,鈥� 鈥渨ill鈥� or 鈥渨ould,鈥� 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; uncertainties as to the timing of and Dyne鈥檚 ability to enroll patients in clinical trials; whether results from preclinical studies and data from clinical trials will be predictive of the final results of the clinical trials or other trials; whether data from clinical trials will support submission for regulatory approvals; uncertainties as to the FDA鈥檚 and other regulatory authorities鈥� interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and as to the regulatory approval process for Dyne鈥檚 product candidates; whether Dyne鈥檚 cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses, debt service obligations and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne鈥檚 filings with the Securities and Exchange Commission (SEC), including the company鈥檚 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鈥檚 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鈥檚 views as of any date subsequent to the date of this press release.
Contacts:
Investors
Mia Tobias
781-317-0353
Media
Stacy Nartker聽
781-317-1938
FAQ
What is the significance of FDA Breakthrough Therapy Designation for Dyne Therapeutics (DYN) DYNE-251?
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