Duchene Muscular Dystrophy Pipeline Drugs Report 2025: A Detailed Landscape of Therapeutic Candidates, Clinical Trials Progress, and Outlook

DelveInsight's, "Duchene Muscular Dystrophy Pipeline Insight 2025" report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in Duchene Muscular Dystrophy pipeline landscape. It covers the Duchene Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Duchene Muscular Dystrophy pipeline 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 Duchene Muscular Dystrophy Pipeline Report

* On 16 September 2025, 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 greater than or equal to 8 to < 18 years old receiving corticosteroid therapy.
* On 11 September 2025, Taiho Pharmaceutical Co., Ltd . organized a study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy. The main purpose of this cohort is to assess the efficacy of TAS-205 in patients with Duchenne muscular dystrophy (DMD) compared with placebo as measured by the mean change from baseline to 52 weeks in the time to rise from the floor
* On 10 September 2025, NS Pharma Inc. initiated a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
* On 10 September 2025, REGENXBIO Inc. announced a clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
* DelveInsight's Duchene Muscular Dystrophy Pipeline report depicts a robust space with 75+ active players working to develop 75+ pipeline therapies for Duchene Muscular Dystrophy treatment.
* The leading Duchene 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 Duchene Muscular Dystrophy Therapies such as Vamorolone, Sevasemten 10 mg, Ataluren, CRD007, Givinostat, RGX-202, DS-5141b, SGT-003 , and others.

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The Duchene Muscular Dystrophy Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Duchene Muscular Dystrophy Pipeline Report also highlights the unmet needs with respect to the Duchene Muscular Dystrophy.

Duchene Muscular Dystrophy Overview

Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. It is the most common hereditary neuromuscular disease and does not exhibit a predilection for any race or ethnic group. Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness. This weakness may present initially with difficulty in ambulation but progressively advances to such an extent that affected patients are unable to carry out activities of daily living and must use wheelchairs. Cardiac and orthopedic complications are common, and death usually occurs in the twenties due to respiratory muscle weakness or cardiomyopathy. Current therapy is centered on treatment with glucocorticoids and physiotherapy to prevent orthopedic complications.

Duchene Muscular Dystrophy Emerging Drugs Profile

* 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).

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Duchene 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.

The Duchene Muscular Dystrophy Pipeline report provides insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Duchene 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 Duchene Muscular Dystrophy Treatment.
* Duchene Muscular Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Duchene 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 Duchene Muscular Dystrophy market.

Duchene Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

* Oral
* Intravenous
* Subcutaneous

Duchene 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 Duchene Muscular Dystrophy Pipeline Report

* Coverage- Global
* Duchene 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.
* Duchene Muscular Dystrophy Therapies- Vamorolone, Sevasemten 10 mg, Ataluren, CRD007, Givinostat, RGX-202, DS-5141b, SGT-003 , and others.
* Duchene Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Duchene Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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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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