Duchenne Muscular Dystrophy: Opportunity Analysis and Forecast to 2033

Description

This report covers the 7MM (US, France, Germany, Italy, Spain, UK, Japan) and provides an Excel-based forecast model for the DMD market through 2033.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. Dystrophin is part of a group of proteins, which together form the dystrophin-associated protein complex (DPC). These proteins form a transmembrane scaffold that is crucial for the structure of the muscle tissue, protecting muscle fibers from injury during muscle contraction and relaxation. DMD is an X-linked recessive disorder mainly affecting males, with female carriers.

The DMD market across the 7MM was valued at $2.3 billion in 2023. Over the 10-year forecast period, the market is projected to grow at a CAGR of 8.5%, reaching $5.2 billion by 2033. The major driver for this growth will be the launch of several pipeline agents: Roche's gene therapy, Capricor's cell therapy deramiocel, PTC Therapeutics' ataluren in the US, and other first-in-class therapies.

Key Highlights

Report deliverables include a Pdf and Excel-based forecast model
Forecasts includes the 7MM
Forecasts covers from 2023-2033

Scope

Overview of DMD, including epidemiology, symptoms, diagnosis, and disease management.
Annualized DMD treatment market revenue, cost of therapy per patient, and treatment usage patterns forecast from 2023 to 2033.
Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications of these factors for the DMD therapeutics market.
Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for DMD treatment. The most promising candidates in Phase III and Phase IIb development are profiled.
Analysis of the current and future market competition in the global DMD therapeutics market. Insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide qualitative analysis of its implications.
Pipeline analysis: Emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.

Reasons to Buy

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
Develop business strategies by understanding the trends shaping and driving the 7MM DMD therapeutics market.
Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the 7MM DMD therapeutics market in the future.
Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Product Code: GDHCOA038

1. Preface

1.1. Contents
1.2. Abbreviations
1.3. Related Reports

2. Executive Summary

3. Disease Overview

3.1. Overview of Duchenne Muscular Dystrophy
3.2. Classification of DMD
3.3. Etiology of DMD
3.4. Pathophysiology of DMD
3.5. Diagnostic Paradigm
3.6. DMD SWOT Analysis

4. Epidemiology

4.1. Diagnosed Incident Cases of DMD, Men, Ages 0-39 Years, 2023-33
4.2. Diagnosed Prevalent Cases of DMD, Men, Ages 0-39 Years, 2023-33
4.3. Age-Specific Trends in Diagnosed Prevalent Cases of DMD, Men, 2023
4.4. Diagnosed Prevalent Cases of DMD by Severity, Men, Ages 0-39 Years, 2023
4.5. Diagnosed Prevalent Cases of DMD with a Specific Mutation, Men, Ages 0-39 Years, 2023
4.6. Diagnosed Prevalent Cases of DMD with Large Mutation Subtypes, Men, Ages 0-39 Years, 2023
4.7. Diagnosed Prevalent Cases of DMD with Small Mutation Subtypes, Men, Ages 0-39 Years, 2023
4.8. Diagnosed Prevalent Cases of DMD by Applicability of Therapies, Men, Ages 0-39 Years, 2023
4.9. Sources Used to Forecast Diagnosed Incident Cases of DMD
4.10. Sources Used to Forecast Diagnosed Prevalent Cases of DMD
4.11. Sources Used to Forecast Diagnosed Prevalent Cases of DMD by Severity
4.12. Sources Used to Forecast Diagnosed Prevalent Cases of DMD with a Specific Mutation, Diagnosed Prevalent Cases of DMD with Large Mutation Subtypes, Diagnosed Prevalent Cases of DMD with Small Mutation Subtypes
4.13. Sources Used to Forecast Diagnosed Prevalent Cases of DMD by Applicability of Therapies
4.14. Sources and Methodology - Diagnosed Incident Cases and Diagnosed Prevalent Cases of DMD
4.15. Sources and Methodology - Diagnosed Prevalent Cases of DMD by Severity, Specific Mutation, and Applicability of Therapies

5. Current Treatment Options

5.1. Treatment Paradigm
5.2. Current Treatments for DMD
5.3. Product Profile: Exondys 51 (eteplirsen)
5.4. Product Profile: Amondys 45 (casimersen)
5.5. Product Profile: Vyondys 53 (golodirsen)
5.6. Product Profile: Viltepso (viltolarsen)
5.7. Product Profile: Exon-Skipping Therapies
5.8. Product Profile: Emflaza (deflazacort)
5.9. Product Profile: Agamree (vamorolone)
5.10. Product Profile: Prednisolone
5.11. Product Profile: Steroids
5.12. Product Profile: Duvyzat (givinostat hydrochloride)
5.13. Product Profile: Elevidys (delandistrogene moxeparvovec-rokl)
5.14. Product Profile: Gene Therapy
5.15. Product Profile: Translarna (ataluren)
5.16. Product Profile: Translarna

6. Unmet Needs and Opportunities

6.1. Unmet Needs in DMD
6.2. Delay or Reverse Disease Regression
6.3. Access to Treatments for Non-ambulatory Patients
6.4. Cost of Therapies

7. R&D Strategies

7.1. Trends in Clinical Trial Design in DMD (1/2)
7.2. Trends in Clinical Trial Design in DMD (2/2)
7.3. Trends in Deal-Making in DMD
7.4. Top 10 Deals for DMD

8. Pipeline Assessment

8.1. DMD Pipeline Overview
8.2. Late-Stage Pipeline Agents for DMD
8.3. Product Profile: NS Pharma's Viltolarsen
8.4. Product Profile: NS Pharma's Brogidirsen
8.5. Product Profile: Italfarmaco's Givinostat
8.6. Product Profile: Roche/Sarepta Therapeutics' delandistrogene moxeparvovec (SRP-9001)
8.7. Product Profile: Gene Therapy
8.8. Product Profile: PTC Therapeutics' Translarna (ataluren)
8.9. Product Profile: Capricor's Deramiocel
8.10. Product Profile: Taiho Pharmaceutical's Pizuglanstat (TAS-205)
8.11. Product Profile: Edgewise Therapeutic's Sevasemten
8.12. Clinical Trials in the DMD Space