Duchenne Muscular Dystrophy Pipeline: 75+ Leading Companies Driving Innovation in Next-Generation Therapies | DelveInsight

The Duchenne Muscular Dystrophy market is rapidly advancing and is fueled by groundbreaking research and innovative therapies from companies such as Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, and Edgewise Therapeutics. These industry pioneers are transforming treatment strategies and redefining the future of Duchenne Muscular Dystrophy, bringing new hope to patients worldwide.

DelveInsight's "Duchenne Muscular Dystrophy Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Duchenne Muscular Dystrophy market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Duchenne Muscular Dystrophy drugs, the Duchenne Muscular Dystrophy pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
• DelveInsight's Duchenne Muscular Dystrophy Pipeline analysis depicts a robust space with 75+ active players working to develop 75+ pipeline drugs for Duchenne Muscular Dystrophy treatment.
• The leading Duchenne Muscular Dystrophy companies include Santhera Pharmaceuticals, Sarepta Therapeutics, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Daiichi Sankyo, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and others are evaluating their lead assets to improve the Duchenne Muscular Dystrophy treatment landscape.
• Key Duchenne Muscular Dystrophy pipeline therapies in various stages of development include Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, and others.
• In March 2025, Capricor Therapeutics announced the FDA has accepted for review its Biologics License Application seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy cardiomyopathy.
• In March 2025, the FDA accepted Capricor Therapeutics' application for approval of deramiocel, a cell therapy developed to treat heart muscle disease in individuals with Duchenne muscular dystrophy, and granted it priority review to accelerate the agency's decision.
• In January 2025, IPS HEART received Orphan Drug Designation from the FDA for GIVI-MPCs, recognizing their potential to create new muscle with full-length dystrophin in Becker Muscular Dystrophy. The therapy has shown promising results in generating human muscle with full-length dystrophin in dystrophic pigs, young and aged Duchenne Muscular Dystrophy mice, and a sarcopenia mouse model.
• In January 2025, Capricor Therapeutics announced the submission of its Biologics License Application to the FDA for full approval of deramiocel, an investigational cell therapy for treating Duchenne muscular dystrophy cardiomyopathy.
• In November 2024, Regenxbio is advancing its gene therapy RGX-202 for Duchenne muscular dystrophy into pivotal development, with plans for a BLA submission by 2026. The company has secured FDA alignment on an accelerated approval pathway, proposing RGX-202 as a one-time treatment, potentially challenging Sarepta's Elevidys in the DMD market.
• In November 2024, Cumberland Pharmaceuticals Inc. announced that the FDA granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy. The company is currently conducting the FIGHT DMDTM trial, a Phase II, multicenter, double-blind, placebo-controlled study to evaluate the pharmacokinetics, safety, and efficacy of once-daily oral Ifetroban in DMD patients. Results are expected later this year.

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Duchenne Muscular Dystrophy Overview
Duchenne muscular dystrophy (DMD) is a severe, inherited neuromuscular disorder and the most prevalent hereditary form of muscular dystrophy. It affects individuals across all races and ethnicities. The condition is caused by mutations in the dystrophin gene, resulting in progressive degeneration and weakening of muscle fibers. Early signs often include difficulty walking, which worsens over time and eventually leads to loss of mobility and dependence on a wheelchair. Complications involving the heart and skeletal system are common, and most patients succumb in their twenties due to respiratory failure or cardiomyopathy. Current treatment primarily involves glucocorticoids and physical therapy to slow disease progression and manage orthopedic issues.

Dystrophin, a large cytoskeletal protein, is essential for maintaining the integrity of muscle cells. Found in both muscle and non-muscle tissues, dystrophin forms part of the dystrophin-glycoprotein complex (DGC), which links the muscle cell cytoskeleton to the extracellular matrix. In DMD, the absence of dystrophin and associated DGC proteins leads to increased membrane instability, disrupted calcium regulation, and oxidative stress, all of which contribute to muscle cell damage and necrosis. As the disease progresses, the ability of muscle to regenerate declines, and damaged muscle tissue is gradually replaced by connective and fat tissue.

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Duchenne Muscular Dystrophy Treatment Analysis: Drug Profile
Vamorolone: Santhera
Vamorolone is a first-in-class investigational therapy designed to bind the same receptors as corticosteroids but modify downstream receptor activity. This unique mechanism may separate the drug's efficacy from the common safety concerns associated with traditional steroids, offering a potentially safer alternative for children and adolescents with Duchenne muscular dystrophy (DMD). Given the significant side effects of long-term corticosteroid use, vamorolone could address a key unmet need in this population. Santhera acquired worldwide rights to vamorolone in DMD and other indications from ReveraGen on September 2, 2020, and the companies aimed to complete their rolling NDA submission to the U.S. FDA by June 2022.

Givinostat: Italfarmaco
Givinostat is an HDAC inhibitor being developed for both Duchenne and Becker muscular dystrophies (DMD and BMD). Unlike therapies targeting specific mutations, givinostat works on downstream pathogenetic processes, making it a potential treatment for a broader patient population. Its mechanism of action aims to counteract disease-related events across all muscle groups, regardless of the specific genetic mutation.

Key Duchenne Muscular Dystrophy Therapies and Companies
• Vamorolone: Santhera
• Givinostat: Italfarmaco
• Pamrevlumab: Fibrogen
• Pizuglanstat (TAS-205): Taiho Pharmaceutical
• SRP-5051: Sarepta Therapeutics
• Allogeneic Cardiosphere-derived Cells (CAP-1002): Capricor
• ITF2357 (Givinostat): Italfarmaco

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Duchenne Muscular Dystrophy Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Intravenous
• Subcutaneous

By Molecule Type
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Duchenne Muscular Dystrophy Pipeline Report
• Coverage: Global
• Key Duchenne Muscular Dystrophy Companies: Santhera Pharmaceuticals, Sarepta Therapeutics, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Daiichi Sankyo, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, and others.
• Key Duchenne Muscular Dystrophy Pipeline Therapies: Vamorolone, Delandistrogene moxeparvovec, Givinostat, WVE N531, Pamrevlumab, EDG 5506, Fordadistrogene movaparvovec, Renadirsen, SRP 5051, EN 001, TAS-205, SGT 001, CAP 1002, NS 089/NCNP 02, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Duchenne Muscular Dystrophy Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Duchenne Muscular Dystrophy Pipeline Therapeutics
6. Duchenne Muscular Dystrophy Pipeline: Late-Stage Products (Phase III)
7. Duchenne Muscular Dystrophy Pipeline: Late-Stage Products (Phase III)
8. Duchenne Muscular Dystrophy Pipeline: Mid-Stage Products (Phase II)
9. Duchenne Muscular Dystrophy Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

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