Duchenne Muscular Dystrophy (DMD) Therapeutics Market to Grow by USD 4.37 Billion from 2024-2028, Driven by High Unmet Needs and AI-Powered Market Evolution - Technavio

NEW YORK, Nov. 6, 2024 /PRNewswire/ -- Report on how AI is driving market transformation - The global duchenne muscular dystrophy (DMD) therapeutics market  size is estimated to grow by USD 4.37 billion from 2024-2028, according to Technavio. The market is estimated to grow at a CAGR of  28.21%  during the forecast period. High unmet need for duchenne muscular dystrophy therapeutics is driving market growth, with a trend towards strong pipeline for duchenne muscular dystrophy therapeutics. However, high treatment costs of duchenne muscular dystrophy  poses a challenge.Key market players include BioMarin Pharmaceutical Inc., Capricor Therapeutics Inc., CRISPR Therapeutics AG, CVS Health Corp., Daiichi Sankyo Co. Ltd., Editas Medicine Inc., Eli Lilly and Co., F. Hoffmann La Roche Ltd., FibroGen Inc., Italfarmaco Holding SPA, Nippon Shinyaku Co. Ltd., Otsuka Holdings Co. Ltd., Pfizer Inc., PTC Therapeutics Inc., Santhera Pharmaceuticals Holding AG, Sarepta Therapeutics Inc., The Cleveland Clinic Foundation, Vertex Pharmaceuticals Inc., and Wave Life Sciences Ltd..

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Key Market Trends Fueling Growth

Several prominent pharmaceutical companies are actively engaged in the development of novel therapeutics for Duchenne Muscular Dystrophy (DMD), a progressive muscle-wasting condition with limited treatment options. The significant unmet need in this indication, driven by its debilitating nature, has fueled research and development efforts. Vendors are exploring various approaches, including antisense oligonucleotide-based therapy, gene therapy, cell therapy, biologics, and small molecule-based drugs. In February 2021, Sarepta Therapeutics secured FDA approval for AMONDYS 45 (casimersen) injection for DMD patients amenable to skipping Exon 45. Additionally, three therapeutic candidates are in Phase III clinical trials, and two are in Phase II/III trials. For instance, Pfizer Inc. Initiated the Phase 3 CIFFREO study in January 2021 to evaluate the efficacy and safety of its investigational gene therapy candidate, PF-06939926, for boys with DMD. These advancements present substantial opportunities for the DMD therapeutics market during the forecast period.

The Duchenne Muscular Dystrophy (DMD) therapeutics market is witnessing significant growth due to the introduction of innovative treatments like Translarna, Tabrecta, and Elamipretide. These molecular-based therapies, including exon skipping drugs and gene replacement therapies, are particularly effective for ambulatory children with nonsense mutations. Exon skipping, a popular approach, corrects the genetic error by skipping over problematic sections of the DMD gene. However, stringent regulatory procedures ensure only safe and effective treatments reach the market. For instance, Translarna, Tabrecta, and Elamipretide have shown promising clinical efficacy in improving muscle function and reducing disease progression. The steroid therapy segment continues to dominate the market but is expected to face stiff competition from these advanced treatments.

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Market Challenges

• Duchenne Muscular Dystrophy (DMD) is a rare and debilitating genetic disorder that primarily affects males, leading to muscle weakness and eventual loss of mobility. This progressive condition results from the insufficient production of functional dystrophin protein, a crucial component for maintaining muscle structure. By age ten, most patients lose the ability to walk, and the disorder can lead to premature death due to respiratory and heart failure. The high cost of available DMD therapies, such as EXONDYS 51 and Emflaza, poses a significant economic burden on patients and their families. The annual cost for EXONDYS 51 treatment is estimated at approximately USD300,000, while Emflaza costs USD35,000 for male patients weighing up to 25 kg, with costs increasing as weight rises. This financial burden can lead to decreased patient adherence, resulting in both volume and value erosion in the DMD therapeutics market. Consequently, the high cost of treatments may hinder market growth during the forecast period.
• The Duchenne Muscular Dystrophy (DMD) therapeutics market is experiencing significant growth due to the introduction of innovative treatments like Translarna, Tabrecta, and Elamipretide. These molecular-based therapies, specifically exon skipping drugs, focus on addressing nonsense mutations in ambulatory children. However, the market faces challenges due to stringent regulatory procedures and the need for clinical efficacy. Exon skipping and gene replacement therapies are promising, but their long-term benefits and safety profiles require further investigation. Steroid therapy remains a mainstay treatment, but its limitations in addressing the disease's progression necessitate the exploration of new therapeutic approaches. The market's future relies on the successful development and approval of these novel treatments.

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Segment Overview 

This duchenne muscular dystrophy (dmd) therapeutics market report extensively covers market segmentation by

1. Type 
• 1.1 Biologics
• 1.2 Small molecules
2. Distribution Channel
• 2.1 Offline
• 2.2 Online
3. Geography 
• 3.1 North America
• 3.2 Europe
• 3.3 Asia
• 3.4 Rest of World (ROW)

1.1 Biologics-  The Duchenne Muscular Dystrophy (DMD) therapeutics market is experiencing significant growth due to the increasing number of diagnosed cases and ongoing research for effective treatments. Companies are investing in developing innovative therapies, such as gene therapies and exon skipping treatments, to address the underlying genetic cause of DMD. Strategic collaborations and partnerships are also driving advancements in this field. The market is expected to continue expanding, providing opportunities for both established and emerging players.

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Research Analysis

Duchenne Muscular Dystrophy (DMD) is a progressive genetic disease characterized by muscle weakness and wasting. The DMD therapeutics market is witnessing significant advancements with the emergence of novel molecular-based therapies, including exon-skipping drugs like Translarna (ataluren) and Tabrecta, and mutation-targeted therapies such as Elamipretide. Orphan Drug Designation has accelerated the development of these innovative treatments. Gene therapy advancements, including fordadistrogene movaparvovec and gene replacement therapies, are also showing promise. Collaborations between research organizations, academic institutions, and key players are driving the market's growth. The market is segmented into molecular-based therapies (exon-skipping drugs, steroid therapy, myostatin inhibition, and gene therapy), hospital pharmacies, and the steroid therapy segment holds a significant share. The DMD treatment market is expected to grow sustainably due to investment pockets in strategic business decisions, with locations like Portland, Oregon, at the forefront of research and development. Despite stringent regulatory procedures and clinical efficacy challenges, the market's future looks bright with ongoing research and advancements in mutation-specific therapies.

Market Research Overview

Duchenne Muscular Dystrophy (DMD) is a progressive and debilitating genetic disorder affecting approximately 1 in every 3,500 live births. The disease is caused by a nonsense mutation in the dystrophin gene, leading to the production of a truncated and non-functional protein. The DMD therapeutics market is segmented into molecular-based therapies, including exon skipping drugs and gene replacement therapies. Exon skipping, such as Translarna and SpliceCorrect, aims to restore the production of a functional dystrophin protein by skipping over the mutated exon. The steroid therapy segment, which includes prednisone and deflazacort, remains the mainstay of treatment, but molecular-based therapies are gaining prominence. The development of these therapies is subject to stringent regulatory procedures to ensure clinical efficacy, with Tabrecta (eteplirsen) and Elamipretide being notable examples of recent approvals for specific patient populations, namely ambulatory children.

Table of Contents:

1 Executive Summary
2 Market Landscape
3 Market Sizing
4 Historic Market Size
5 Five Forces Analysis
6 Market Segmentation

• Type
• Biologics
• Small Molecules
• Distribution Channel
• Offline
• Online
• Geography
• North America
• Europe
• Asia
• Rest Of World (ROW)

7 Customer Landscape
8 Geographic Landscape
9 Drivers, Challenges, and Trends
10 Company Landscape
11 Company Analysis
12 Appendix

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SOURCE Technavio