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Duchenne Muscular Dystrophy – Opportunity Assessment and Forecast to 2030

The duchenne muscular dystrophy (DMD) market across the US and Japan was valued at $619.0 million in 2020. The market is projected to grow at a CAGR of more than 11% during the forecast period 2021-2030. The diagnosed prevalent cases of DMD are expected to increase in the US and decrease in Japan between 2020 to 2030. In 2020, the most prevalent mutation among DMD cases across both markets was deletion mutation. The least prevalent mutation type in both markets was the splice site mutation.

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin. The disease is an X linked recessive disorder mainly affecting males, with female carriers. While females are generally unaffected, some may show a milder phenotype (“manifesting carriers”) such as cardiac changes, mild muscle weakness, and muscle cramping. Lack of dystrophin in muscle cells causes them to be fragile and easily damaged DMD is progressive and most affected individuals require a wheelchair by adolescence. Serious life-threatening conditions including cardiomyopathy and respiratory difficulties ultimately develop and lead to mortality of DMD patients between 19 and 25 years of age.

Overview of the DMD market

Overview of the DMD market

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What are the market dynamics in the DMD market?

The US represents more than 90% of the DMD sales in 2020, but its contribution is expected to decrease in 2030. The dominance of the US is due to its larger DMD population coupled with the higher prices of drugs compared to Japan. Additionally, available DMD treatments in Japan are only Viltepso and highly genericized prednisone, in contrast to the US where there are four exon-skipping products and two steroids, namely Emflaza and prednisone (off-label).

Large unmet needs and strong advocacy by patients and parents will expedite review processes and push novel treatments to market. DMD disease market forecasts that pipeline drugs will acquire almost 60% of the market in 2030, impacting a three-fold decrease in the market share of exon-skipping therapies and shrinking that of steroids by half. The major driver of DMD market growth will be the launch of eight new pipeline agents, the high cost of therapy for gene and cell-based therapies, and the anticipated increase in treatment rates throughout the forecast period.

What are key segments in the DMD market?

The key drug classes segments in the DMD disease market are steroids, exon-skipping therapies, and pipeline drugs. In 2020, exon-skipping therapies and steroids dominated the DMD disease market.

Steroids in the duchenne muscular dystrophy market

Steroids can treat all DMD patient segments, regardless of mutation types. They fall under the gold standards of care and are widely accepted by physicians for treating DMD. Steroids such as Emflaza and Prednisone will continue to have a place in the treatment paradigm and will limit the competition from pipeline agents.

Exon-skipping therapies in the duchenne muscular dystrophy market

Over the past five years, the FDA has also approved a new treatment named exon-skipping therapies. These products include Exondys 51 (for patients with mutations amenable to exon-51 skipping), Vyondys 53 (for patients with mutations amenable to exon-53 skipping), Viltepso (for patients with mutations amenable to exon-53 skipping), and Amondys 45 (for patients with mutations amenable to exon-45 skipping) for DMD patients. These products are antisense oligonucleotides that skip the mutated area of the dystrophin gene. This allows cells to join a different set of exons together to produce a protein that is shorter than usual but may have some function. These treatments have a better safety and tolerability profile as compared to glucocorticoids.

Pipeline drugs in the duchenne muscular dystrophy market

Pipeline drug classes like gene therapies and Capricor’s cell therapy are expected to have a significant effect on the market in the coming days. Pipeline drugs like fordadistrogene movaparvovec have a wider scope of amenable mutations than exon-skipping therapies. They target both skeletal and cardiac muscles and can benefit cardiac and pulmonary functions in patients.

DMD market, by key segments

DMD market, by key segments

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Which are the key market players in the DMD Market?

The key market vendors in the DMD market are Sarepta Therapeutics Inc, Nippon Shinyaku Co Ltd, PTC Therapeutics Inc, Asahi Kasei Pharma Corp, Koa Isei So Ltd, Italfarmaco SpA, FibroGen Inc, Taiho Pharmaceutical Co Ltd, Capricor Therapeutics Inc, Santhera Pharmaceuticals Holding AG, and Pfizer Japan Inc.

DMD market, by key players

DMD market, by key players

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Market report scope

Market value (Year – 2020) $619.0 million
Growth rate (CAGR) >11%
Base year estimation 2020
Forecast period 2021-2030
Segmentation Steroids, Exon-skipping Therapies, and Pipeline Drugs
Key market players Sarepta Therapeutics Inc, Nippon Shinyaku Co Ltd, PTC Therapeutics Inc, Asahi Kasei Pharma Corp, Koa Isei So Ltd, Italfarmaco SpA, FibroGen Inc, Taiho Pharmaceutical Co Ltd, Capricor Therapeutics Inc, Santhera Pharmaceuticals Holding AG, Pfizer Japan Inc.
Countries covered The US and Japan

Scope

  • Overview of DMD including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Topline DMD market revenue, the annual cost of therapy, and major pipeline product sales in the forecast period.
  • Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting DMD therapeutics sales in the US and Japan.
  • Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the US and Japanese DMD market. Insightful review of key industry drivers, restraints, and challenges.

Key Highlights

Diagnosed prevalent cases of DMD are expected to increase by 0.14% in the US and decrease by 1.27% in Japan between 2020 and 2030.

Current treatments in the US and Japan consist of exon-skipping therapies and steroids.

Greatest unmet needs in the DMD space pertain to disease progression rate, access to treatment for specific patient segments, and cost of therapies.

The DMD pipeline consists of 23 drugs across all stages of development in the US and Japan, with 8 products in late-stage development.

The DMD market in the US and Japan was valued at $619.0M in 2020. The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 12.0%, reaching $1.9B by 2030.

Reasons to Buy

  • Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current deals and technologies to identify companies with the highest potential.
  • Develop business strategies by understanding the trends shaping and driving the DMD pharma market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the DMD pharma market in the future.
  • Design your development strategy through a review of potential novel targets or combinations in the DMD space.
  • Understand the challenges and strategies impacting the development of therapeutic agents in DMD preclinical studies and clinical trials.
  • Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the classes of therapies and indications that present maximum opportunities for consolidations, investments, and strategic partnerships in the DMD space.

Key Players

Sarepta Therapeutics

Nippon Shinyaku

PTC Therapeutics

Asahi Kasei Pharma

Koa Isei Pharma

Pfizer

Italfarmaco

FibroGen

Taiho Pharmaceutical

Santhera Pharmaceuticals

Capricor Therapeutics

Table of Contents

Table of Contents (PowerPoint Deck)

1 Executive Summary

1.1 Executive Summary

2 Disease Overview

2.1 Overview of Eosinophilic Esophagitis

2.2 Pathophysiology of DMD

2.3 Diagnostic Paradigm

2.4 DMD SWOT Analysis

3 Epidemiology

3.1 Diagnosed Prevalent Cases of DMD, Boys/Men, Ages 0–39 Years, 2020–2030

3.2 Diagnosed Prevalent Cases of DMD by Mutation, Boys/Men, Ages 0–39 Years, 2020

3.3 Sources and Methodology for Diagnosed Prevalence of DMD

3.4 Sources and Methodology

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profile: Exondys 51 (eteplirsen)

4.4 Product Profile: Amondys 45 (casimersen)

4.5 Product Profile: Vyondys 53 (golodirsen)

4.6 Product Profile: Viltepso (viltolarsen)

4.7 Product Profile: Emflaza (deflazacort)

4.8 Product Profile: prednisolone

5 Unmet Needs and Opportunities

5.1 Unmet Needs in DMD

5.1 Delay or Reverse Disease Regression

5.2 Access to Treatments for Non-ambulatory Patients

5.3 Cost of Therapies

6 Pipeline Assessment

6.1 DMD Pipeline Overview

6.2 Clinical Trial Mapping of Late-Stage DMD Pipeline Drugs

6.3 Late-Stage Pipeline Agents

6.4 Product Profile: PTC Therapeutics’ Translarna (ataluren)

6.5 Product Profile: Pfizer’s fordadistrogene movaparvovec

6.6 Product Profile: Italfarmaco’s givinostat

6.7 Product Profile: FibroGen’s pamrevlumab

6.8 Product Profile: Taiho Pharmaceutical’s pizuglanstat

6.9 Product Profile: Sarepta Therapeutics’ delandistrogene moxeparvovec

6.10 Product Profile: Santhera Pharmaceuticals’ vamorolone

6.11 Product Profile: Capricor Therapeutics’ CAP-1002

7 R&D Strategies

7.1 Trends in Clinical Trial Design in DMD

7.2 Trends in Deal-Making in DMD

7.3 Trends in Clinical Trial Design in DMD

8 Market Outlook

8.1 DMD Disease Market Forecast

8.2 Market Drivers and Barriers

9 Appendix

9.1 Bibliography

9.2 Primary Research: KOL Information

9.3 About the Authors

10 Contact Us

Frequently Asked Questions

The duchenne muscular dystrophy market across the US and Japan was valued at $619.0 million in 2020.

The market is projected to grow at a CAGR of more than 11% during the period 2021-2030.

By drug classes, the DMD market has been segmented into steroids, exon-skipping therapies, and pipeline drugs.

The key players in the DMD market are Sarepta Therapeutics Inc, Nippon Shinyaku Co Ltd, PTC Therapeutics Inc, Asahi Kasei Pharma Corp, Koa Isei So Ltd, Italfarmaco SpA, FibroGen Inc, Taiho Pharmaceutical Co Ltd, Capricor Therapeutics Inc, Santhera Pharmaceuticals Holding AG, and Pfizer Japan Inc.

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