Dublin, May 08, 2025 (GLOBE NEWSWIRE) -- The "Duchenne Muscular Dystrophy - Pipeline Insight, 2025" clinical trials has been added to ResearchAndMarkets.com's offering.
This report provides comprehensive insights about 75+ companies and 75+ pipeline drugs in Duchenne Muscular Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Report Highlights
Companies and academics are working to assess challenges and seek opportunities that could influence R&D Duchenne Muscular Dystrophy. The therapies under development are focused on novel approaches to treat/improve Duchenne Muscular Dystrophy.
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.
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).
Duchenne Muscular Dystrophy: Therapeutic Assessment
This segment of the report provides insights about the Duchenne Muscular Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Duchenne Muscular Dystrophy
There are approx. 75+ key companies which are developing the therapies Duchenne Muscular Dystrophy. The companies which have their Duchenne Muscular Dystrophy drug candidates in the most advanced stage, i.e phase III include Roche
Phases
The report covers around 75+ products under different phases of clinical development like
- 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
Route of Administration
Duchenne 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
Molecule Type
Products have been categorized under various Molecule types such as
- Small molecule
- Cell Therapy
- Peptides
- Polymer
- Small molecule
- Gene therapy
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Pipeline Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Duchenne Muscular Dystrophy therapeutic drugs key players involved in developing key drugs.
Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Duchenne Muscular Dystrophy drugs.
Key Questions
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Duchenne Muscular Dystrophy drugs?
- How many Duchenne Muscular Dystrophy drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Duchenne Muscular Dystrophy?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Duchenne Muscular Dystrophy and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- 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
Key Products
- 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
- UX 810
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/7x02f9
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