The global duchenne muscular dystrophy drugs market size is expected to reach USD 9.91 billion by 2030, according to a new report by Grand View Research, Inc., expanding at a CAGR of 16.8% from 2024 to 2030. Several factors such as the emergence of mutation-specific therapies, growing target population, and favorable government initiatives are driving the market.
The DMD therapeutics market has only three approved products - Exondys51, Translarna, and Emflaza. Increasing uptake of mutation-targeted therapies is likely to boost the sales of branded drugs. However, stringent regulatory procedures and lack of standardized protocol for the determination of clinical efficacy are key challenges for the market.
Exon-skipping platform is estimated to hold about 45.0% of the market share by 2023, driven by increasing adoption of Exondys51 and impending approval of golodirsen and casimersen. Mutation-specific therapies, such as Translarna, are anticipated to face limited adoption due to premium pricing. Associated adverse effects of steroid therapy make them a less favored treatment option.
The DMD drugs market is projected to become intensely competitive in medium- to long-term. The sector has witnessed delays and denials of several key products by the U.S. FDA due to insufficient trials. Raxone (by Santhera) and Givinostat (by Italfarmaco) remain the most strategically significant R&D pipeline assets for the DMD market, with a good possibility of regulatory approval during the forecast period.
Launch of late-stage pipeline products is poised to alter the DMD therapeutic landscape in the near future. Several novel mechanisms of action, such as NF-κB inhibition, myostatin inhibition, and gene therapy are under investigation. Other drug classes, such as exon-skipping and mutation-suppression, are expected to gain traction over the forecast period, supported by the rising adoption of these therapeutics.
Milo Biotechnology is one of the first companies to venture into gene therapy for DMD. The company’s investigational therapy uses an adeno-associated virus as a myostatin inhibitor and is in the early stages of development. Wave Life Science’s pipeline product is likely to perform 25 times better in terms of clinical efficacy than the existing exon-skipping drugs.
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The molecular based therapies segment dominated the market in 2023 with a share of 42.0% due to the rising prevalence of DMD disease and rising preference for molecular based therapies as they directly treat the genetic root cause of DMD.
Hospital pharmacies dominated the market with a share of 41.4% in 2023 due to the rise in drug availability and therapies for treating DMD disease.
North America Duchenne muscular dystrophy (DMD) drugs market dominated with a market share of 45.0% in 2023 attributed to the presence of major healthcare institutes and the rise in the number of patients suffering from DMD.
Grand View Research has segmented the global Duchenne muscular dystrophy drugs market on the basis of treatment, distribution channel, and region:
Duchenne Muscular Dystrophy Drugs Treatment Outlook (Revenue, USD Million, 2018 - 2030)
Molecular Based Therapies
Mutation Suppression
Exon Suppression
Steroid Based Therapies
Non-steroidal Anti-Inflammatory Drugs (NSAIDs)
Others
Duchenne Muscular Dystrophy Drugs Distribution Channel Outlook (Revenue, USD Million, 2018 - 2030)
Hospital Pharmacies
Retail Pharmacies
Online Pharmacies
Duchenne Muscular Dystrophy Drugs Regional Outlook (Revenue, USD Million, 2018 - 2030)
North America
U.S.
Canada
Mexico
Europe
UK
Germany
France
Italy
Spain
Denmark
Sweden
Norway
Asia Pacific
Japan
China
India
Australia
South Korea
Thailand
Latin America
Brazil
Argentina
Middle East and Africa (MEA)
South Africa
Saudi Arabia
UAE
Kuwait
List of Key Players of Duchenne Muscular Dystrophy Drugs Market
Aurobindo Pharma
Capricor Therapeutics, Inc.
Catalyst Pharmaceuticals, Inc.
EspeRare Foundation
FibroGen, Inc.
ITALFARMACO S.p.A.
NS Pharma, INC.
PTC Therapeutics.
Santhera Pharmaceuticals
Sarepta Therapeutics, Inc.
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