Duchenne Muscular Dystrophy - Pipeline Insight, 2024

This 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 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.

Geography Covered

• Global coverage

Duchenne Muscular Dystrophy Understanding

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.

Dystrophin is a large cytoskeletal protein that facilitates interactions between the cytoskeleton, cell membrane, and extracellular matrix. It is located at the plasma membrane in both muscle and non-muscle tissues. Dystrophin is a critical part of the dystrophin-glycoprotein complex (DGC), which plays an important role as being a structural unit of muscle. In DMD, both dystrophin and DGC proteins are missing, leading to excessive membrane fragility and permeability, dysregulation of calcium homeostasis, oxidative damage. These factors play a crucial role in muscle cell necrosis. As patients with DMD age, the regenerative capacity of the muscles appears to be exhausted, and connective and adipose tissue gradually replaces muscle fibers.

Duchenne Muscular Dystrophy - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Duchenne Muscular Dystrophy pipeline landscape is provided which includes the disease overview and Duchenne Muscular Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Duchenne Muscular Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Duchenne Muscular Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The 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. 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).

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

This 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.

Duchenne Muscular Dystrophy: Pipeline Development 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.

Pipeline 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.

Duchenne Muscular Dystrophy Report Insights

• Duchenne Muscular Dystrophy Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Duchenne Muscular Dystrophy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

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
• UX 810

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

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