Comprehensive Therapeutic Development Pipeline for Duchenne Muscular Dystro

DelveInsight's latest analysis of the "Duchenne Muscular Dystrophy Pipeline Intelligence 2025" delivers extensive insights on more than 75 organizations and over 75 developmental compounds in the Duchenne Muscular Dystrophy therapeutic landscape. The analysis encompasses drug candidate profiles across both clinical and preclinical development phases. Additionally, it includes evaluation of Duchenne Muscular Dystrophy investigational treatments by formulation type, developmental phase, administration method, and molecular classification. The report also identifies dormant pipeline candidates in this therapeutic area.

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Essential Highlights from the Duchenne Muscular Dystrophy Pipeline Analysis

On December 11, 2025- Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical-stage biopharmaceutical organization advancing innovative therapies in oncology and other high-need disease areas, revealed that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (DMD).

On December 09, 2025- Solid Biosciences Inc. revealed an investigation to examine the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this investigation. Cohort 1 will include participants 4 to < 7 years of age. Cohort 2 will include participants 7 to < 12 years of age. Cohort 3 will include participants 0 to < 4 years of age. Cohort 4 will include participants 12 to < 18 years of age. Cohort 5 will include participants 10 to < 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the investigation for 5 total years for long-term follow up.

DelveInsight's Duchenne Muscular Dystrophy pipeline analysis reveals a dynamic field with over 75 active organizations advancing more than 75 pipeline candidates for Duchenne Muscular Dystrophy therapy.

The prominent Duchenne Muscular Dystrophy Companies include Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and additional organizations.

Notable Duchenne Muscular Dystrophy Therapies include Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and additional candidates.

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Duchenne Muscular Dystrophy Background

Duchenne Muscular Dystrophy (DMD) represents a rare, inherited, progressive neuromuscular disorder triggered by mutations in the DMD gene, which is responsible for producing dystrophin—a crucial protein that helps maintain muscle cell integrity. Without dystrophin, muscle fibers become fragile and easily damaged, resulting in ongoing muscle weakness and degeneration. DMD predominantly impacts young boys, with symptoms typically manifesting between 2–5 years of age. Early signs include difficulty running, climbing stairs, frequent falls, enlarged calves, and delayed motor milestones. Over time, the disease advances to affect the skeletal muscles, heart (cardiomyopathy), and lungs, making mobility increasingly challenging. Most individuals require a wheelchair in early adolescence and need respiratory and cardiac support as they grow older. The progressive nature of DMD creates devastating impact on patients, families, and caregivers, while the genetic understanding has opened avenues for innovative therapeutic strategies including gene therapy, exon-skipping approaches, and dystrophin restoration technologies aimed at addressing the fundamental molecular pathology underlying this severe neuromuscular condition.

Duchenne Muscular Dystrophy Emerging Drug Profiles

Vamorolone: Santhera

Vamorolone represents a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to 'dissociate' efficacy from typical steroid safety concerns and therefore could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from patient quality of life. On September 2, 2020, Santhera exercised its option and obtained worldwide rights to vamorolone in Duchenne muscular dystrophy and all other indications. Santhera and ReveraGen expect to complete the rolling NDA submission to the U.S. FDA in June 2022.

Givinostat: Italfarmaco

Givinostat represents an HDAC inhibitor (HDACi), a principle candidate, currently being created for the treatment of DMD and BMD. Since Givinostat acts on the pathogenetic events downstream of the genetic defects, it is potentially a treatment for the whole DMD and BMD population and to counter the disease pathogenetic events in all muscular districts.

Pamrevlumab: Fibrogen

Pamrevlumab represents a first-in-class antibody created by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is advancing towards Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and pancreatic cancer and has been granted Orphan Drug Designation (ODD) in each of these indications, and is currently in a Phase 2 investigation for Duchenne muscular dystrophy (DMD).

The Duchenne Muscular Dystrophy Pipeline Analysis Delivers Intelligence Into

The analysis delivers comprehensive intelligence about organizations that are creating therapies for the treatment of Duchenne Muscular Dystrophy with aggregate therapies created by each organization for the same indication.

It evaluates various therapeutic candidates categorized into early-phase, mid-phase, and late-phase of development for Duchenne Muscular Dystrophy Treatment.

Duchenne Muscular Dystrophy Companies are engaged in targeted therapeutics development with corresponding active and inactive (dormant or terminated) projects.

Duchenne Muscular Dystrophy Drugs in development classified by developmental phase, administration route, target receptor, monotherapy or combination therapy, various mechanisms of action, and molecular classification.

Comprehensive analysis of partnerships (company-company partnerships and company-academia partnerships), licensing agreements and financial arrangements for future progression of the Duchenne Muscular Dystrophy market.

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Duchenne Muscular Dystrophy Companies

Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and additional organizations.

Duchenne Muscular Dystrophy Pipeline Analysis Delivers Therapeutic Evaluation

The pipeline analysis delivers therapeutic evaluation of developmental drugs by Administration Route:

• Oral

• Intravenous

• Subcutaneous

Duchenne Muscular Dystrophy Products Organized by Molecular Classification

Products have been organized under various molecular types including:

• Small molecule

• Cell Therapy

• Peptides

• Polymer

• Gene therapy

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Parameters of the Duchenne Muscular Dystrophy Pipeline Analysis

Coverage: Global

Duchenne Muscular Dystrophy Companies: Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, EDG 5506 Edgewise Therapeutics, Fordadistrogene movaparvovec, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and additional organizations.

Duchenne Muscular Dystrophy Therapies: Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and additional candidates.

Duchenne Muscular Dystrophy Therapeutic Evaluation by Product Type: Mono, Combination, Mono/Combination

Duchenne Muscular Dystrophy Therapeutic Evaluation by Clinical Phases: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Contents Overview

1. Introduction

2. Executive Summary

3. Duchenne Muscular Dystrophy: Overview

4. Pipeline Therapeutics

5. Therapeutic Assessment

6. Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective

7. Late Stage Products (Phase III)

8. Delandistrogene moxeparvovec: Roche

9. Drug profiles in the detailed report…..

10. Mid-Stage Products (Phase II)

11. SRP 5051: Sarepta Therapeutics

12. Drug profiles in the detailed report…..

13. Early Stage Products (Phase I/II)

14. WVE N531: Wave Life Sciences

15. Drug profiles in the detailed report…..

16. Early Stage Products (Phase I)

17. EDG 5506: Edgewise Therapeutics

18. Drug profiles in the detailed report…..

19. Inactive Products

20. Duchenne Muscular Dystrophy Key Companies

21. Duchenne Muscular Dystrophy Key Products

22. Duchenne Muscular Dystrophy- Unmet Needs

23. Duchenne Muscular Dystrophy- Market Drivers and Barriers

24. Duchenne Muscular Dystrophy- Future Perspectives and Conclusion

25. Duchenne Muscular Dystrophy Analyst Views

26. Duchenne Muscular Dystrophy Key Companies

27. Appendix

Conclusion

The Duchenne Muscular Dystrophy pipeline demonstrates extraordinary advancement with diverse therapeutic approaches addressing critical unmet needs in this devastating pediatric neuromuscular disorder. With over 75 organizations developing more than 75 pipeline candidates, the therapeutic landscape is experiencing unprecedented innovation driven by enhanced understanding of dystrophin biology and genetic mechanisms underlying muscle degeneration. The remarkable diversity of therapeutic modalities under investigation—spanning gene therapies, exon-skipping oligonucleotides, gene editing technologies, anti-inflammatory agents, and dystrophin restoration strategies—reflects the multifaceted approach being pursued to address this complex genetic condition from multiple angles.

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