DelveInsight’s “Duchenne Muscular Dystrophy Pipeline Insight 2025” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in the Duchenne Muscular Dystrophy pipeline landscape. It covers the Duchenne Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Duchenne Muscular Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Duchenne Muscular Dystrophy Pipeline Report
- On January 26, 2026- Cumberland Pharmaceuticals conducted a study with an optional open-label extension to determine the safety, pharmacokinetics (PK) and efficacy of two doses of oral ifetroban in subjects with DMD. DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban or placebo once daily for 12 months. Subjects will be enrolled into one of three treatment groups, low-dose ifetroban, high-dose ifetroban or placebo. Each dose level will be evaluated by eight subjects with early stage (LVEF > 45%) DMD-associated cardiomyopathy and eight subjects with more advanced stage (LVEF 35-45%) cardiac disease as there may be differences in the treatment effect based on cardiac involvement. Each subject treated will be evaluated for first-dose and steady-state exposure PK. All subjects who receive treatment will be assessed for safety.
- On January 21, 2026- Italfarmaco conducted a study of GIVINOSTAT in all DMD (Duchenne’s muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- On January 09, 2026- Santhera Pharmaceuticals initiated a study have completed previous studies with vamorolone and continued to receive vamorolone under special programs: Compassionate Use Program [CUP], Named Patient Program [NPP] or Expanded Access Protocol [EAP]. All subjects will continue treatment with vamorolone under Guardian protocol instead. The primary objective of this study is to evaluate the safety of long-term treatment with vamorolone in boys with Duchenne Muscular Dystrophy regarding vertebral fractures. Secondary study objectives will evaluate the safety of long-term treatment with vamorolone on non-vertebral fractures, cataracts, delayed puberty, overall safety as well as ambulatory and non-ambulatory function.
- On January 05, 2026- Hoffmann-La Roche conducted a study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD age ≥ 8 to < 18 years old receiving corticosteroid therapy.
- On January 05, 2026- Satellos Bioscience Inc. announced a global phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. One dose of SAT-3247 and placebo will be studied in the US and Canada; two doses of SAT-3247 and placebo will be studied in UK, EU, Serbia, and Australia.
- On January 02, 2026- Pfizer conducted a study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
- DelveInsight’s Duchenne Muscular Dystrophy pipeline report depicts a robust space with 75+ active players working to develop 75+ pipeline therapies for Duchenne Muscular Dystrophy treatment.
- The leading Duchenne Muscular Dystrophy Companies such as 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 others.
- Promising Duchenne Muscular Dystrophy Therapies such as Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and others.
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Duchenne Muscular Dystrophy Overview
Duchenne Muscular Dystrophy (DMD) is a rare, inherited, progressive neuromuscular disorder caused by mutations in the DMD gene, which is responsible for producing dystrophin—a crucial protein that helps keep muscle cells intact. Without dystrophin, muscle fibers become fragile and easily damaged, leading to ongoing muscle weakness and degeneration. DMD typically affects young boys, with symptoms usually appearing 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 progresses 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.
Duchenne Muscular Dystrophy Emerging Drugs
- Vamorolone: Santhera
Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors1,2. 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, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed 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 is a first-in-class antibody developed 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 trial for Duchenne muscular dystrophy (DMD).
The Duchenne Muscular Dystrophy Pipeline Report Provides Insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Duchenne Muscular Dystrophy with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Duchenne Muscular Dystrophy Treatment.
- Duchenne Muscular Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Duchenne Muscular Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Duchenne Muscular Dystrophy market.
Learn more about Duchenne Muscular Dystrophy Drugs opportunities in our groundbreaking Duchenne Muscular Dystrophy Research and development projects @ Duchenne Muscular Dystrophy Unmet Needs
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 others.
Duchenne Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
- Oral
- Intravenous
- Subcutaneous
Duchenne Muscular Dystrophy Products have been categorized under various Molecule types such as
- Small molecule
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
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Scope of the Duchenne Muscular Dystrophy Pipeline Report
- 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 others.
- Duchenne Muscular Dystrophy Therapies- Vamorolone, Sevasemten 10 mg, Givinostat, DS-5141b, SGT-003, PF-06939926, NS-089/NCNP-02, and others.
- Duchenne Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Duchenne Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
For a detailed overview of our latest research findings and future plans, read the full details of Duchenne Muscular Dystrophy Pipeline on our website @ Duchenne Muscular Dystrophy Drugs and Companies
Table of Contents
- Introduction
- Executive Summary
- Duchenne Muscular Dystrophy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Delandistrogene moxeparvovec: Roche
- Drug profiles in the detailed report…..
- Mid-Stage Products (Phase II)
- SRP 5051: Sarepta Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- WVE N531: Wave Life Sciences
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- EDG 5506: Edgewise Therapeutics
- Drug profiles in the detailed report…..
- Inactive Products
- Duchenne Muscular Dystrophy Key Companies
- Duchenne Muscular Dystrophy Key Products
- Duchenne Muscular Dystrophy- Unmet Needs
- Duchenne Muscular Dystrophy- Market Drivers and Barriers
- Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
- Duchenne Muscular Dystrophy Analyst Views
- Duchenne Muscular Dystrophy Key Companies
- Appendix
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