DelveInsight’s, “Duchenne Muscular Dystrophy Pipeline Insight 2024” report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in 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
- In September 2024:- Edgewise Therapeutics Inc.- A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker.
- In September 2024:- Solid Biosciences Inc.- This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study. Cohort 1 will include participants 4 to
- 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 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).
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 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
Discover the latest advancements in Duchenne Muscular Dystrophy Treatment by visiting our website. Stay informed about how we’re transforming the future of musculoskeletal @ Duchenne Muscular Dystrophy Market Drivers and Barriers, and Future Perspectives
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 Content
- 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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