Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1
Avidity Biosciences (RNA) has achieved a significant milestone as its drug delpacibart etedesiran (del-desiran) received Orphan Drug designation in Japan for treating myotonic dystrophy type 1 (DM1). This marks the first DM1 treatment to receive this designation in Japan.
Del-desiran, designed to target the root cause of DM1 (a progressive and often fatal neuromuscular disease), has already secured multiple regulatory designations including Breakthrough Therapy, Orphan Drug, and Fast Track from the FDA, along with Orphan designation from the EMA.
The company reports promising results from the MARINA and MARINA-OLE studies, showing favorable long-term safety, disease progression reversal, and sustained clinical improvements. Avidity expects to complete enrollment in the Phase 3 HARBOR trial by mid-2025, with marketing applications planned for submission starting 2026 in the U.S., EU, and Japan.
Avidity Biosciences (RNA) ha raggiunto un traguardo significativo poiché il suo farmaco delpacibart etedesiran (del-desiran) ha ricevuto la designazione di Farmaco Orfano in Giappone per il trattamento della distrofia miotonica di tipo 1 (DM1). Questo segna il primo trattamento per la DM1 a ricevere questa designazione in Giappone.
Il del-desiran, progettato per affrontare la causa principale della DM1 (una malattia neuromuscolare progressiva e spesso fatale), ha già ottenuto diverse designazioni regolatorie tra cui Terapia Innovativa, Farmaco Orfano e Percorso Veloce dalla FDA, insieme alla designazione di Farmaco Orfano dall'EMA.
L'azienda riporta risultati promettenti dagli studi MARINA e MARINA-OLE, mostrando una sicurezza a lungo termine favorevole, una reversibilità della progressione della malattia e miglioramenti clinici sostenuti. Avidity prevede di completare l'arruolamento nella fase 3 dello studio HARBOR entro metà 2025, con le domande di marketing programmate per la presentazione a partire dal 2026 negli Stati Uniti, nell'UE e in Giappone.
Avidity Biosciences (RNA) ha logrado un hito significativo ya que su medicamento delpacibart etedesiran (del-desiran) recibió la designación de Medicamento Huérfano en Japón para el tratamiento de la distrofia miotónica tipo 1 (DM1). Esto marca el primer tratamiento para DM1 en recibir esta designación en Japón.
El del-desiran, diseñado para atacar la causa raíz de DM1 (una enfermedad neuromuscular progresiva y a menudo fatal), ya ha asegurado múltiples designaciones regulatorias, incluyendo Terapia Innovadora, Medicamento Huérfano y Vía Rápida de la FDA, junto con la designación de Medicamento Huérfano de la EMA.
La empresa informa resultados prometedores de los estudios MARINA y MARINA-OLE, mostrando una seguridad a largo plazo favorable, reversión de la progresión de la enfermedad y mejoras clínicas sostenidas. Avidity espera completar la inscripción en el ensayo de fase 3 HARBOR para mediados de 2025, con aplicaciones de comercialización previstas para su presentación a partir de 2026 en EE. UU., UE y Japón.
아비디티 바이오사이언스(Avidity Biosciences, RNA)는 약물 델파시바트 에테데시란(delpacibart etedesiran)이 일본에서 희귀의약품으로 지정되었다는 중요한 이정표를 달성했습니다. 이는 일본에서 DM1(근긴장성 근육병) 치료제로서 이 지정을 받은 첫 번째 사례입니다.
DM1의 근본 원인을 목표로 설계된 델-데시란은 이미 FDA로부터 혁신 치료제, 희귀의약품, 신속 심사 등 여러 규제 지정을 확보했으며, EMA로부터도 희귀의약품 지정을 받았습니다.
회사는 MARINA 및 MARINA-OLE 연구에서 장기적인 안전성, 질병 진행의 역전 및 지속적인 임상 개선을 보여주는 유망한 결과를 보고했습니다. 아비디티는 2025년 중반까지 3상 HARBOR 시험의 등록을 완료할 것으로 예상하며, 2026년부터 미국, EU 및 일본에서 마케팅 신청을 제출할 계획입니다.
Avidity Biosciences (RNA) a atteint une étape significative car son médicament delpacibart etedesiran (del-desiran) a reçu la désignation de Médicament Orphelin au Japon pour le traitement de la dystrophie myotonique de type 1 (DM1). Cela marque le premier traitement de la DM1 à recevoir cette désignation au Japon.
Le del-desiran, conçu pour cibler la cause profonde de la DM1 (une maladie neuromusculaire progressive et souvent mortelle), a déjà obtenu plusieurs désignations réglementaires, y compris Thérapie Innovante, Médicament Orphelin et Voie Accélérée de la FDA, ainsi que la désignation de Médicament Orphelin de l'EMA.
La société rapporte des résultats prometteurs des études MARINA et MARINA-OLE, montrant une sécurité à long terme favorable, une inversion de la progression de la maladie et des améliorations cliniques soutenues. Avidity s'attend à terminer l'inscription dans l'essai de phase 3 HARBOR d'ici mi-2025, avec des demandes de commercialisation prévues pour soumission à partir de 2026 aux États-Unis, dans l'UE et au Japon.
Avidity Biosciences (RNA) hat einen bedeutenden Meilenstein erreicht, da sein Medikament delpacibart etedesiran (del-desiran) die Orphan Drug-Bezeichnung in Japan für die Behandlung der myotonen Dystrophie Typ 1 (DM1) erhalten hat. Dies ist die erste DM1-Behandlung, die diese Bezeichnung in Japan erhält.
Del-desiran, das darauf ausgelegt ist, die Ursache von DM1 (eine progressive und oft tödliche neuromuskuläre Erkrankung) anzugehen, hat bereits mehrere regulatorische Bezeichnungen erhalten, darunter Durchbruchtherapie, Orphan Drug und Fast Track von der FDA sowie die Orphan-Bezeichnung von der EMA.
Das Unternehmen berichtet von vielversprechenden Ergebnissen aus den Studien MARINA und MARINA-OLE, die eine langfristige Sicherheit, eine Umkehrung des Krankheitsverlaufs und nachhaltige klinische Verbesserungen zeigen. Avidity erwartet, die Rekrutierung in der Phase-3-Studie HARBOR bis Mitte 2025 abzuschließen, mit geplanten Marktzulassungsanträgen, die ab 2026 in den USA, der EU und Japan eingereicht werden sollen.
- First DM1 treatment to receive Orphan Drug designation in Japan
- Secured multiple regulatory fast-track designations across major markets (FDA, EMA)
- Positive clinical data showing disease progression reversal and sustained improvements
- Clear commercialization timeline with marketing applications planned for 2026
- No current revenue from the drug as it's still in development phase
- Marketing applications not expected until 2026, indicating extended timeline to potential revenue
Insights
Japan's Orphan Drug designation (ODD) for del-desiran represents a significant regulatory milestone for Avidity's lead DM1 candidate. This designation - granted to the first investigational DM1 treatment in Japan - provides substantial advantages in a market where fewer than 50,000 patients suffer from this condition.
The ODD package includes prioritized consultation, reduced fees, tax incentives, and priority review - meaningful benefits that can accelerate del-desiran's path to commercialization in Japan. When combined with the drug's existing designations (Breakthrough Therapy, Orphan Drug, Fast Track from FDA; Orphan designation from EMA), Avidity has secured an optimal regulatory pathway across major markets.
From a regulatory perspective, the alignment with global regulators on the registrational path is particularly noteworthy. This coordination has informed the Phase 3 HARBOR trial design, enabling Avidity to pursue simultaneous submissions across the US, EU, and Japan starting in 2026. This harmonized approach is strategically sound, allowing for potential synchronized approvals and a coordinated global launch.
The favorable long-term safety profile and clinical improvements reported from the MARINA and MARINA-OLE studies likely contributed to securing this designation. For rare disease therapeutics, regulatory authorities place high value on consistent safety data alongside signals of efficacy, especially when addressing conditions with no approved therapies.
This ODD in Japan strengthens Avidity's global regulatory position for del-desiran and demonstrates continued de-risking of their lead program. The designation carries both near-term operational advantages (tax incentives, reduced fees) and long-term strategic value by potentially expediting approval in Japan's notoriously stringent regulatory environment.
Del-desiran targets myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease with no approved therapies - positioning Avidity to potentially capture an untapped market. The company's statement that the drug has demonstrated "reversal of disease progression" and "consistent and durable improvements in multiple clinical endpoints" suggests promising efficacy, though detailed data metrics aren't provided.
The Phase 3 HARBOR trial enrollment completion expected by mid-2025 establishes a clear timeline: potential submissions in 2026 across major markets, suggesting possible commercialization by 2027 if approvals are granted. This timeline gives Avidity approximately two years to build commercial infrastructure and prepare market access strategies.
Avidity's AOC platform (Antibody Oligonucleotide Conjugates) represents a differentiated approach to RNA therapeutics. Success with del-desiran would validate this platform technology, potentially increasing the perceived value of other pipeline candidates leveraging the same technology. For investors, this milestone reinforces confidence in both the lead program's regulatory pathway and the company's ability to execute on global development strategy.
Del-desiran first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan
"This decision by MHLW further reinforces the significant potential of del-desiran to address the root cause of DM1 and the urgent need to bring an approved therapy to the many people impacted by this devastating rare disease in
Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the
About the Phase 3 HARBOR™ Trial
The global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. Patients will be administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the HARBOR trial, visit the HARBOR study website or visit http://www.clinicaltrials.gov and search for NCT06411288.
About the Phase 2 MARINA-OLE™ Study
MARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit http://www.clinicaltrials.gov and search for NCT05479981.
About Del-desiran
Del-desiran, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the
About Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is an underrecognized, autosomal dominantly inherited, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people living with DM1.
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 neuromuscular 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
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: planned marketing applications for del-desiran in the
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: Avidity may not realize any benefit from Orphan Drug designation of del-desiran by global health authorities; the data and results produced in Avidity's ongoing studies of del-desiran as of the most recent respective cutoff dates may not be indicative of final results, may not support a BLA submission, may not be satisfactory to the MHLW and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the MHLW and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of the MARINA-OLE and HARBOR studies; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; legislative, judicial and regulatory developments in
Investor Contact:
Kat Lange
(619) 837-5014
investors@aviditybio.com
Media Contact:
(619) 837-5016
media@aviditybio.com
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SOURCE Avidity Biosciences, Inc.
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