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Duchenne Muscular Dystrophy Pipeline Insight Report | 75+ Companies and 75+ Therapies

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Duchenne Muscular Dystrophy Pipeline Insight Report | 75+ Companies and 75+ Therapies

January 16
18:49 2023
Duchenne Muscular Dystrophy Pipeline Insight Report | 75+ Companies and 75+ Therapies

DelveInsight’s, “Duchenne Muscular Dystrophy Pipeline Insight 2023” 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 Duchenne Muscular Dystrophy clinical trial and nonclinical stage products. It also covers the Duchenne 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.

 

Key takeaways from the Duchenne Muscular Dystrophy Pipeline Report

  • 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 are working in the market include Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, and others are evaluating new drugs for duchenne muscular dystrophy to improve the treatment landscape.
  • Promising Duchenne Muscular Dystrophy pipeline therapies in various stages of development include 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, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.
  • The Duchenne Muscular Dystrophy Companies and academics are working to assess challenges and seek opportunities that could influence R&D Duchenne Muscular Dystrophy. The Duchenne Muscular Dystrophy pipeline therapies under development are focused on novel approaches to treat/improve Duchenne Muscular Dystrophy.

 

Discover the recent advances in Duchenne Muscular Dystrophy Treatment Drugs @ Duchenne Muscular Dystrophy Pipeline Outlook

 

Duchenne 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.

 

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).

 

Find out more about Duchenne muscular dystrophy treatment drugs @ Duchenne Muscular Dystrophy Treatment Landscape

 

Duchenne Muscular Dystrophy Therapeutic Assessment

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.

 

Scope of the Duchenne Muscular Dystrophy Pipeline Report

  • Coverage- Global
  • Duchenne Muscular Dystrophy CompaniesSanthera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, and others
  • Duchenne Muscular Dystrophy Pipeline Therapies- 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, ENTR-601-44, SRP-9001, GNT 0004, SRP-5051, UX810, SGT-003, Exon 53, Exon 45, AVGN7, PGN EDO51, PGN EDODM1, PGN EDO53, PGN EDO44, PGN EDO45, ALT1102, BMN 351, AOC 1044, SRP-9001, GNT 0004, SRP-5053, SRP-5045, SRP-5052, SRP-5044, SRP-5050, SRP-5051, DYNE-251, SGT-003, RGX-202, Elamipretide, and others.
  • Duchenne Muscular Dystrophy Segmentation- Phases, Molecule Type, Mechanism of Action, Route of Administration, Product Type

 

Learn more about the emerging Duchenne Muscular Dystrophy pipeline therapies @ Duchenne Muscular Dystrophy Ongoing Clinical Trials Analysis

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Duchenne Muscular Dystrophy: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Duchenne Muscular Dystrophy– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Delandistrogene moxeparvovec: Roche
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. SRP 5051: Sarepta Therapeutics
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I/II)
  14. WVE N531: Wave Life Sciences
  15. Drug profiles in the detailed report…..
  16. Early Stage Products (Phase I)
  17. EDG 5506: Edgewise Therapeutics
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Duchenne Muscular Dystrophy Key Companies
  21. Duchenne Muscular Dystrophy Key Products
  22. Duchenne Muscular Dystrophy- Unmet Needs
  23. Duchenne Muscular Dystrophy- Market Drivers and Barriers
  24. Duchenne Muscular Dystrophy- Future Perspectives and Conclusion
  25. Duchenne Muscular Dystrophy Analyst Views
  26. Duchenne Muscular Dystrophy Key Companies
  27. Appendix

 

Dive deep into rich insights for new drugs for Duchenne Muscular Dystrophy treatment, visit @ Duchenne Muscular Dystrophy Key Companies and Therapies

 

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