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Duchenne Muscular Dystrophy Pipeline, Clinical Trial Analysis and NDA Approvals | 75+ Companies and 75+ Therapies

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Duchenne Muscular Dystrophy Pipeline, Clinical Trial Analysis and NDA Approvals | 75+ Companies and 75+ Therapies

January 17
16:53 2023
Duchenne Muscular Dystrophy Pipeline, Clinical Trial Analysis and NDA Approvals | 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 trials 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.

 

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.
  • Key Duchenne muscular dystrophy companies such as 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.

 

Recent Developmental Activities in the Duchenne Muscular Dystrophy Treatment Landscape

  • In July 2022, Capricor Therapeutics dosed the first subject in Phase III HOPE-3 clinical trial of cell therapy, CAP-1002, to treat late-stage Duchenne muscular dystrophy (DMD).
  • In June 2022, FibroGen announced that it had completed the target enrollment for its ongoing Phase III LELANTOS-2 clinical trial (NCT04632940) of pamrevlumab, a first-in-class connective tissue growth factor (CTGF) inhibitor antibody, in patients with Duchenne muscular dystrophy (DMD). The trial is set to read out topline data in the second half of 2023 after completing the target enrollment of 73 patients with Duchenne muscular dystrophy aged 6 to 12 years.
  • In May 2022, Stealth BioTherapeutics announced that the FDA had granted orphan drug designation to elamipretide for the treatment of Duchenne muscular dystrophy.
  • In January 2022, The US Food and Drug Administration (FDA) approved Regenxbio’s request to launch a Phase I/II clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy (DMD).
  • In June 2020, Sarepta Therapeutics announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs).
  • In November 2021, CANbridge Pharmaceuticals announced that it has entered into a two-year sponsored research agreement with the University of Washington School of Medicine in Seattle, Washington, for gene therapy research in Duchenne muscular dystrophy (DMD), a rare neuromuscular disease

 

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

 

Duchenne Muscular Dystrophy Overview

Duchenne muscular dystrophy 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 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).

 

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 Duchenne Muscular Dystrophy Companies which have their Duchenne Muscular Dystrophy drug candidates in the most advanced stage, i.e phase III include Roche.

 

Duchenne Muscular Dystrophy Therapeutics Assessment

The Duchenne Muscular Dystrophy pipeline report proffers an integral view of the Duchenne muscular dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

 

Scope of the Duchenne Muscular Dystrophy Pipeline Report

  • Coverage- Global
  • Duchenne Muscular Dystrophy Companies- 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.
  • 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 Clinical Trials

 

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

 

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?

 

About Us

DelveInsight is a Business Consulting and Market research company, providing expert business solutions for the healthcare domain and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.

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