The Duchenne muscular dystrophy (DMD) market across the seven major markets (7MM*) is set to grow from $2.3 billion in 2023 to $5.2 billion in 2033, driven by the recent approvals of innovative therapies such as Sarepta Therapeutics and Roche’s Elevidys (delandistrogene moxeparvovec), and Santhera Pharmaceuticals’ Agamree (vamorolone), according to GlobalData, a leading data and analytics company.
GlobalData’s latest report, “Duchenne Muscular Dystrophy Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2033,” reveals that a substantial portion of this market growth is attributed to the treatment of ambulatory DMD patients. Exon-skipping therapies currently dominate the DMD therapeutic landscape, generating approximately $1.0 billion in sales in the 7MM in 2023.
Notably, the sales are derived solely from the US and Japan markets, as exon-skipping therapies have yet to receive regulatory approval in the European Union (EU). Should these therapies gain EU approval by 2033, GlobalData forecasts their contribution to rise to $1.8 billion across the 7MM, a significant market share partly driven by the high annual cost of therapy, which exceeds $1.0 million in the US.
Asiyah Nawab, Healthcare Analyst at GlobalData, comments: “The DMD treatment landscape is evolving with the emergence of novel therapies such as exon-skipping and gene therapies. However, gene therapies in particular, compared to exon-skipping, will have less of an impact due to the small patient share eligible for treatment, in addition to the high cost of these medicines limiting patient’s access. By 2033, GlobalData forecasts gene therapies to contribute $821 million to the DMD market, a lower figure relative to exon-skipping therapies.”
The US is set to remain the dominant market for DMD, accounting for 84.8% of total market share in 2023. This is driven by its rapid adoption of advanced therapies, strong regulatory support, and significant investment in DMD research and treatment.
Regulatory developments have also shaped the market, with Translarna (ataluren) facing challenges in Europe. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has confirmed its recommendation not to renew the conditional marketing authorization for Translarna, citing unconfirmed effectiveness in treating DMD. However, in the US, PTC Therapeutics has resubmitted its New Drug Application (NDA) for Translarna, which the FDA has accepted for review. If approved, Translarna is projected to generate $185 million in US sales alone.
Nawab continues: “Despite advancements, unmet needs remain a critical concern, particularly for non-ambulatory patients. While recent approvals have expanded treatment options for ambulatory individuals, therapeutic availability for non-ambulatory patients remains a key challenge. Many emerging therapies, including exon-skipping and gene therapies, primarily target early-stage or ambulatory patients, leaving a significant gap for those with advanced disease. This, coupled with high treatment costs and regulatory hurdles, underscores the urgent need for more accessible and effective therapies for later-stage DMD patients.”
