Exondys 51 Market Report 2025: Epidemiology, Pipeline Analysis, Market Insights & Forecasts

This Exondys 51 market report delivers an in-depth analysis of the market’s key characteristics, including size, growth potential, and segmentation. It provides a detailed breakdown of the market across major regions and leading countries, highlighting historical data and future growth projections. The report also examines the competitive landscape, market share insights, emerging trends, and strategic developments shaping the market.

The growth observed in the historical period can be attributed to factors such as increased awareness and diagnosis of Duchenne muscular dystrophy (DMD), supportive government policies, a rise in the prevalence of rare diseases, and the growing incidence of Duchenne muscular dystrophy.

The expected growth in the forecast period is driven by the increasing burden of DMD, the growing number of new drug launches, heightened research and development activities, and the rising prevalence of DMD. Key trends during this period include advancements in diagnostic technologies, progress in mutation-targeted therapies, development of mutation-suppressing therapies, ongoing R&D efforts for new drug treatments, and innovations in gene therapies.

The rising burden of genetic disorders is expected to significantly drive the growth of the exondys 51 market. Genetic disorders, which are caused by mutations or abnormalities in an individual’s DNA, are becoming more prevalent due to advancements in medical technology, improvements in diagnosis, and growing awareness. Exondys 51 is a key treatment for genetic disorders such as Duchenne muscular dystrophy (DMD), a severe condition characterized by muscle degeneration. By enabling exon 51 skipping in the dystrophin gene, Exondys 51 helps produce a functional, albeit shortened, dystrophin protein, which slows the progression of muscle degeneration in DMD patients. For example, in July 2024, the National Health Service (NHS) reported that around 70,000 individuals in the UK are affected by muscular dystrophy, with Duchenne being the most common form, further emphasizing the need for effective treatments such as exondys 51.

Rising healthcare investments are expected to propel the exondys 51 market. As healthcare investments increase, driven by factors such as the aging population and growing demand for chronic disease management, these funds enable advancements in medical research and clinical trials. Investment in exon-skipping technologies, which Exondys 51 relies on, is crucial for the continued development and accessibility of treatments for conditions such as Duchenne muscular dystrophy. For example, in May 2024, healthcare spending in the UK grew by 5.6% from 2022 to 2023, which indicates the growing financial support for healthcare innovations. These investments help drive the development of more effective treatments, making them available to a broader patient population.

A key trend in the exondys 51 market is the expanding regulatory approvals for Duchenne muscular dystrophy treatments. Regulatory approvals from health authorities such as the FDA or EMA are critical in determining a drug’s marketability and availability. Recent approvals for gene therapies targeting specific genetic mutations in Duchenne muscular dystrophy, such as Sarepta Therapeutics' Elevidys and Amondys 45, indicate a shift towards personalized treatments. The expanded approval of these therapies offers hope for a wider range of DMD patients, which is crucial for improving accessibility and treatment outcomes. This trend in regulatory approvals will further stimulate the growth of the Exondys 51 market by allowing it to be used for a broader spectrum of genetic mutations and increasing its global availability.

The key company operating in the exondys 51 market is Sarepta Therapeutics.

North America was the largest region in the exondys 51 market in 2024. Europe is expected to be the fastest-growing region in the forecast period. The regions covered in exondys 51 report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the exondys 51 market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Exondys 51 (eteplirsen) is a medication used to treat Duchenne muscular dystrophy (DMD) by promoting exon-skipping of the dystrophin gene, enabling the production of a functional but shortened dystrophin protein. It works by skipping over exon 51 of the dystrophin gene, which allows for the creation of a shorter yet functional version of the dystrophin protein.

The primary indications for exondys 51 include relapsing-remitting multiple sclerosis (RRMS), secondary progressive multiple sclerosis (SPMS), and other forms of multiple sclerosis. RRMS is the most common type of multiple sclerosis, marked by distinct relapses or attacks of neurological symptoms. Exondys 51 is distributed through hospital pharmacies, specialty pharmacies, and online pharmacies, and is used for subcutaneous injection, long-term disease management, and prophylactic treatment.

The exondys 51 market research report is one of a series of new reports that provides exondys 51 market statistics, including exondys 51 industry global market size, regional shares, competitors with an exondys 51 market share, detailed exondys 51 market segments, market trends and opportunities, and any further data you may need to thrive in the exondys 51 industry. This exondys 51 market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The exondys 51 market consists of sales of products including golodirsen (Vyondys 53), viltolarsen (Viltepso), and casimersen (Amondys 45). Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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