The Duchenne Muscular Dystrophy therapies under development are focused on novel approaches to treat/improve the disease condition. Major players such as Sarepta Therapeutics, Pfizer, Santhera Pharmaceuticals, Taiho Pharmaceuticals, and others are involved in developing therapies for Duchenne Muscular Dystrophy. The launch of emerging therapies will significantly impact the Duchenne Muscular Dystrophy market.
DelveInsight’s “Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast 2034” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Duchenne Muscular Dystrophy market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Duchenne Muscular Dystrophy market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Duchenne Muscular Dystrophy Overview
Duchenne Muscular Dystrophy is a severe, genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys, with an incidence of about 1 in 3,500 to 1 in 6,000 male births. DMD is caused by mutations in the DMD gene, which provides instructions for making a protein called dystrophin. Dystrophin is crucial for maintaining muscle fibers’ structural integrity; its absence or significant reduction leads to muscle cell damage and death.
Duchenne Muscular Dystrophy Market Key Facts
- The Duchenne muscular dystrophy market size in the 7MM was around USD 2,150 million in 2023. The market is anticipated to witness a substantial positive shift owing to better uptake of existing drugs, the expected market launch of one-time gene therapies, and raised awareness.
- The main therapeutic strategies for the treatment of Duchenne muscular dystrophy include gene replacement or other genetic therapies linked to specific mutations to restore dystrophin production, membrane stabilization and/or upregulation of compensatory proteins and reduction of the inflammatory cascade and/or enhancement of muscle regeneration.
- The current US market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy.
- In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat Duchenne muscular dystrophy patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany.
- ELEVIDYS, the first FDA-approved gene therapy for Duchenne muscular dystrophy, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta’s gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process.
- In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting Duchenne muscular dystrophy patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan the only approved treatment is VILTEPSO (viltolarsen).
- Expected Launch of potential therapies by key players like, Pfizer (PF-06939926), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), Capricor (CAP-1002), Italfarmaco (Givinostat), Antisense Therapeutics (ATL1102), and Sarepta Therapeutics (SRP-5051), may increase the market size in the coming years, assisted by an increase in the prevalent population of Duchenne muscular dystrophy. It is expected that these therapies will help thrive the market of Duchenne muscular dystrophy post-launch, in the United States. Furthermore, these novel therapies will mark there entry in EU4 and the UK and Japan in subsequent years.
Duchenne Muscular Dystrophy Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Duchenne Muscular Dystrophy market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Duchenne Muscular Dystrophy market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Duchenne Muscular Dystrophy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Duchenne Muscular Dystrophy Epidemiology, Segmented as –
Total Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the 7MM
Age-specific Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the 7MM
Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in the 7MM
Mutation-specific Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the 7MM
Ambulatory and non-ambulatory Population of Duchenne Muscular Dystrophy (DMD) in the 7MM
Duchenne Muscular Dystrophy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the rate of uptake of the potential drugs recently launched in the Duchenne Muscular Dystrophy market or expected to be launched during the study period. The analysis covers the Duchenne Muscular Dystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Duchenne Muscular Dystrophy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Learn How the Duchenne Muscular Dystrophy Market Will Evolve and Grow by 2034 @ https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-market
Leading Companies in the Duchenne Muscular Dystrophy Therapeutics Market Include:
Antisense Therapeutics
Bioleaders Corporation
Capricor
Daiichi Sankyo
FibroGen
Italfarmaco
Mitobridge
Nippon Shinyaku
Pfizer
PTC Therapeutics
ReveraGen BioPharma
Santhera Pharmaceuticals
Sarepta Therapeutics
Solid Biosciences
Taiho Pharmaceutical
Vertex Pharmaceuticals
WaVe life Sciences
And Many Others
Duchenne Muscular Dystrophy Marketed and Emerging Therapies Covered in the Report Include:
Vyondys 53 (Golodirsen): Sarepta Therapeutics
Emflaza: PTC Therapeutics
Exondys 51: Sarepta Therapeutics
Translarna: PTC Therapeutics
Amondys 45: Sarepta Therapeutics
Viltepso: Nippon Shinyaku
PF-06939926: Pfizer
Vamorolone: Santhera Pharmaceuticals/ReveraGen BioPharma
Pizuglanstat (TAS-205): Taiho Pharmaceutical
Givinostat: Italfarmaco
CRISPR/Cas9 gene editing therapy: Vertex Pharmaceuticals
WVE N531: WaVe life Sciences
Pamrevlumab: FibroGen
TAS-205: Taiho Pharmaceutical
MA-0211: Mitobridge
BLS-M22: Bioleaders Corporation
CAP 1002: Capricor
DS-5141: Daiichi Sankyo
And Many More
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @ https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-market
Table of Contents
1. Key Insights
2. Executive Summary
3. Duchenne Muscular Dystrophy Competitive Intelligence Analysis
4. Duchenne Muscular Dystrophy Market Overview at a Glance
5. Duchenne Muscular Dystrophy Disease Background and Overview
6. Duchenne Muscular Dystrophy Patient Journey
7. Duchenne Muscular Dystrophy Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Duchenne Muscular Dystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Duchenne Muscular Dystrophy Unmet Needs
10. Key Endpoints of Duchenne Muscular Dystrophy Treatment
11. Duchenne Muscular Dystrophy Marketed Products
12. Duchenne Muscular Dystrophy Emerging Drugs and Latest Therapeutic Advances
13. Duchenne Muscular Dystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Duchenne Muscular Dystrophy Market Outlook (In US, EU5, and Japan)
16. Duchenne Muscular Dystrophy Access and Reimbursement Overview
17. KOL Views on the Duchenne Muscular Dystrophy Market
18. Duchenne Muscular Dystrophy Market Drivers
19. Duchenne Muscular Dystrophy Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
Download the Sample PDF to Learn More About the Key Offerings of the Report @ https://www.delveinsight.com/sample-request/duchenne-muscular-dystrophy-market
The Duchenne muscular dystrophy market size in the 7MM was around USD 2,150 million in 2023. The market is anticipated to witness a substantial positive shift owing to better uptake of existing drugs, the expected market launch of one-time gene therapies, and raised awareness.The main therapeutic strategies for the treatment of Duchenne muscular dystrophy include gene replacement or other genetic therapies linked to specific mutations to restore dystrophin production, membrane stabilization and/or upregulation of compensatory proteins and reduction of the inflammatory cascade and/or enhancement of muscle regeneration.The current US market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy.In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat Duchenne muscular dystrophy patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany.ELEVIDYS, the first FDA-approved gene therapy for Duchenne muscular dystrophy, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta’s gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process.In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting Duchenne muscular dystrophy patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan the only approved treatment is VILTEPSO (viltolarsen).Expected Launch of potential therapies by key players like, Pfizer (PF-06939926), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), Capricor (CAP-1002), Italfarmaco (Givinostat), Antisense Therapeutics (ATL1102), and Sarepta Therapeutics (SRP-5051), may increase the market size in the coming years, assisted by an increase in the prevalent population of Duchenne muscular dystrophy. It is expected that these therapies will help thrive the market of Duchenne muscular dystrophy post-launch, in the United States. Furthermore, these novel therapies will mark there entry in EU4 and the UK and Japan in subsequent years.
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