Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics (Nasdaq: DYN) has received European Commission orphan drug designation for DYNE-251, its investigational therapy for Duchenne muscular dystrophy (DMD). The designation follows promising long-term clinical data presented in March 2025, showing sustained functional improvement through 18 months in the Phase 1/2 DELIVER trial.
The trial evaluates DYNE-251 in DMD patients amenable to exon 51 skipping. The company has completed enrollment of 32 patients in the registrational expansion cohort, with data expected in late 2025. Dyne plans to submit a Biologics License Application for US accelerated approval in early 2026.
The EU orphan designation provides benefits including reduced regulatory fees, clinical protocol assistance, and potential 10-year market exclusivity. DYNE-251 previously received FDA orphan drug and rare pediatric disease designations in March 2023.
Dyne Therapeutics (Nasdaq: DYN) ha ottenuto la designazione di farmaco orfano dalla Commissione Europea per DYNE-251, la sua terapia sperimentale per la distrofia muscolare di Duchenne (DMD). Questa designazione segue dati clinici a lungo termine promettenti presentati a marzo 2025, che mostrano un miglioramento funzionale sostenuto per 18 mesi nello studio di Fase 1/2 DELIVER.
Lo studio valuta DYNE-251 in pazienti con DMD idonei al salto dell’esone 51. L’azienda ha completato il reclutamento di 32 pazienti nella coorte di espansione per la registrazione, con dati previsti per la fine del 2025. Dyne prevede di presentare una domanda di autorizzazione biologica per l’approvazione accelerata negli USA all’inizio del 2026.
La designazione di farmaco orfano nell’UE offre vantaggi come la riduzione delle tasse regolatorie, assistenza nel protocollo clinico e potenziale esclusività di mercato per 10 anni. DYNE-251 aveva già ricevuto le designazioni di farmaco orfano e malattia pediatrica rara dalla FDA a marzo 2023.
Dyne Therapeutics (Nasdaq: DYN) ha recibido la designación de medicamento huérfano por parte de la Comisión Europea para DYNE-251, su terapia en investigación para la distrofia muscular de Duchenne (DMD). Esta designación sigue a datos clínicos a largo plazo prometedores presentados en marzo de 2025, que muestran una mejora funcional sostenida durante 18 meses en el ensayo de Fase 1/2 DELIVER.
El ensayo evalúa DYNE-251 en pacientes con DMD aptos para el salto del exón 51. La compañía ha completado la inscripción de 32 pacientes en la cohorte de expansión para registro, con datos esperados a finales de 2025. Dyne planea presentar una solicitud de licencia biológica para la aprobación acelerada en EE.UU. a principios de 2026.
La designación de medicamento huérfano en la UE ofrece beneficios como reducción de tasas regulatorias, asistencia en el protocolo clínico y posible exclusividad de mercado por 10 años. DYNE-251 ya había recibido las designaciones de medicamento huérfano y enfermedad pediátrica rara por la FDA en marzo de 2023.
Dyne Therapeutics (나스닥: DYN)이(가) 유럽연합 집행위원회로부터 DYNE-251에 대해 희귀의약품 지정을 받았습니다. DYNE-251은 뒤쉔 근이영양증(DMD) 치료를 위한 연구 중인 치료제입니다. 이 지정은 2025년 3월에 발표된 장기 임상 데이터에 따른 것으로, 1/2상 DELIVER 시험에서 18개월간 지속된 기능 개선이 확인되었습니다.
본 시험은 엑손 51 스킵핑이 가능한 DMD 환자를 대상으로 DYNE-251을 평가합니다. 회사는 등록 확장 코호트에 32명의 환자 등록을 완료했으며, 데이터는 2025년 말에 발표될 예정입니다. Dyne은 2026년 초 미국에서 가속 승인용 생물의약품 허가 신청을 계획하고 있습니다.
EU 희귀의약품 지정은 규제 수수료 감면, 임상 프로토콜 지원, 최대 10년간 시장 독점권 부여 등의 혜택을 제공합니다. DYNE-251은 2023년 3월 FDA로부터 희귀의약품 및 희귀 소아 질환 지정도 받은 바 있습니다.
Dyne Therapeutics (Nasdaq : DYN) a obtenu la désignation de médicament orphelin de la Commission européenne pour DYNE-251, sa thérapie expérimentale contre la dystrophie musculaire de Duchenne (DMD). Cette désignation fait suite à des données cliniques à long terme prometteuses présentées en mars 2025, montrant une amélioration fonctionnelle soutenue pendant 18 mois dans l’essai de phase 1/2 DELIVER.
L’essai évalue DYNE-251 chez des patients atteints de DMD éligibles au saut de l’exon 51. L’entreprise a terminé le recrutement de 32 patients dans la cohorte d’expansion d’enregistrement, avec des données attendues fin 2025. Dyne prévoit de soumettre une demande d’autorisation de mise sur le marché biologique pour une approbation accélérée aux États-Unis début 2026.
La désignation de médicament orphelin dans l’UE offre des avantages tels que la réduction des frais réglementaires, l’assistance au protocole clinique et une exclusivité de marché potentielle de 10 ans. DYNE-251 avait déjà reçu les désignations de médicament orphelin et de maladie pédiatrique rare par la FDA en mars 2023.
Dyne Therapeutics (Nasdaq: DYN) hat von der Europäischen Kommission die Orphan-Drug-Zulassung für DYNE-251 erhalten, seine experimentelle Therapie für Duchenne-Muskeldystrophie (DMD). Die Zulassung folgt auf vielversprechende Langzeit-Klinikdaten, die im März 2025 präsentiert wurden und eine anhaltende funktionelle Verbesserung über 18 Monate im Phase-1/2-DELIVER-Studie zeigen.
Die Studie bewertet DYNE-251 bei DMD-Patienten, die für das Überspringen von Exon 51 geeignet sind. Das Unternehmen hat die Einschreibung von 32 Patienten in der registrierenden Erweiterungskohorte abgeschlossen, mit Daten, die Ende 2025 erwartet werden. Dyne plant, Anfang 2026 einen Antrag auf Zulassung eines Biologikums für eine beschleunigte Zulassung in den USA einzureichen.
Die EU-Orphan-Drug-Zulassung bietet Vorteile wie reduzierte regulatorische Gebühren, Unterstützung bei klinischen Protokollen und eine mögliche 10-jährige Marktexklusivität. DYNE-251 erhielt bereits im März 2023 Orphan-Drug- und seltene pädiatrische Krankheitsdesignationen von der FDA.
- Received EU orphan drug designation for DYNE-251
- Demonstrated sustained functional improvement through 18 months in clinical trials
- Successfully completed enrollment of 32 patients in registrational expansion cohort
- Potential for 10-year market exclusivity in EU upon approval
- Multiple regulatory designations secured (EU orphan, FDA orphan, rare pediatric disease)
- Final trial data not available until late 2025
- Regulatory approvals still pending and not guaranteed
Insights
EMA orphan drug designation boosts DYNE-251's regulatory profile, offering market exclusivity and validating Dyne's DMD clinical progress.
The European Commission's orphan drug designation for DYNE-251 represents a significant regulatory milestone for Dyne Therapeutics in their Duchenne muscular dystrophy (DMD) program. This designation provides substantial benefits including reduced regulatory fees, protocol assistance, and potential 10-year market exclusivity in the European Union upon approval. This builds upon the previously secured FDA orphan drug and rare pediatric disease designations from March 2023, creating a comprehensive regulatory advantage package across major markets.
The company has established a clear regulatory timeline with data from the fully enrolled 32-patient registrational expansion cohort expected in late 2025 and BLA submission for US accelerated approval planned for early 2026. Particularly notable is the acceptance of Stride Velocity 95th Centile (SV95C) as a primary endpoint for European DMD trials, which positions Dyne favorably for potential EU approval.
The presented data showing "unprecedented and sustained functional improvement" through 18 months of treatment is especially significant in the DMD therapeutic landscape. DMD is a devastating genetic disorder characterized by progressive muscle weakness, and the reported functional improvements through SV95C measurement provide objective evidence that may support European regulatory approval. This orphan designation reinforces the regulatory pathway for DYNE-251 in exon 51 skipping-amenable DMD patients, representing an important advancement in the company's global regulatory strategy.
Dyne's DYNE-251 shows unprecedented sustained functional improvement in DMD patients, with orphan designation boosting its development pathway.
The EMA orphan drug designation for DYNE-251 marks an important development for patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The designation validates the scientific approach and potential clinical impact of this therapy in a devastating disease with treatment options.
What's particularly noteworthy in the clinical data presented is the sustained functional improvement through 18 months of treatment. In DMD therapeutics, where slowing disease progression is typically the primary goal, demonstrated functional improvement represents a potentially significant advancement. The use of Stride Velocity 95th Centile (SV95C) as an assessment tool is especially relevant, as it's accepted as a primary endpoint for European regulatory approval.
The fully enrolled registrational expansion cohort of 32 patients will provide critical data in late 2025, potentially supporting regulatory submissions beginning in early 2026. This timeline suggests confidence in the therapeutic potential of DYNE-251. The company's focus on exon 51 skipping addresses a specific genetic subset of DMD patients, offering a targeted therapeutic approach.
This orphan designation, combined with the existing FDA orphan and rare pediatric disease designations, establishes a favorable regulatory framework across major markets. For patients with this progressive and ultimately fatal condition, the reported unprecedented functional improvements could translate to meaningful clinical benefit if confirmed in the larger cohort data expected later this year.
- Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months -
- Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 -
WALTHAM, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile (SV95C), an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe.
“Our recent long-term DELIVER trial results demonstrated clinically relevant and sustained functional improvement through 18 months, including as assessed by SV95C, which may support a strong rationale for regulatory approval in Europe,” said Doug Kerr, MD, PhD, chief medical officer of Dyne. “We are pleased that the EC has granted orphan drug designation to DYNE-251, reinforcing our belief that our next-generation exon 51 skipping investigational therapy for DMD may be able to bring clinically meaningful functional improvement to those living with this devastating disease. With full enrollment of the registrational expansion cohort in the DELIVER trial complete, we look forward to sharing data from this cohort in late 2025 and the potential to move forward with our first regulatory submissions in early 2026.”
The EC grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union (EU). Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and the potential for up to 10 years of market exclusivity in the EU if approved. DYNE-251 was also granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations in March 2023.
Key Milestones for the DELIVER Trial
- Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping.
- Dyne has fully enrolled the registrational expansion cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025.
- Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.
About the DELIVER Trial
DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who 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 every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit clinicaltrials.gov (NCT05524883) and euclinicaltrials.eu (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 people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) 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 and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping.
In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.
About Duchenne Muscular Dystrophy (DMD)
DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. 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 no cure for DMD, and currently approved therapies provide limited benefit.
About Dyne Therapeutics
Dyne Therapeutics is 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 deliver to muscle 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 therapeutic potential of DYNE-251, the anticipated timeline for reporting additional data from the DELIVER clinical trial, the availability of expedited approval pathways for DYNE-251, expectations regarding the timing and outcome of interactions with regulatory authorities, and expectations regarding the timing of submitting applications for U.S. Accelerated Approval and other regulatory approvals, 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 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’s and other regulatory authorities’ 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’s product candidates; 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 the company’s most recent Form 10-K 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.
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