Spinal Muscular Atrophy Market Report 2025

This Spinal Muscular Atrophy 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 spinal muscular atrophy market size has grown rapidly in recent years. It will grow from $3.53 billion in 2024 to $4.01 billion in 2025 at a compound annual growth rate (CAGR) of 13.5%. The growth during the historic period can be attributed to factors such as improved diagnosis and early detection, the rising prevalence of rare genetic diseases, enhanced healthcare infrastructure, government and regulatory support, and a growing demand for addressing unmet healthcare needs.

The spinal muscular atrophy market size is expected to see rapid growth in the next few years. It will grow to $6.59 billion in 2029 at a compound annual growth rate (CAGR) of 13.2%. For the forecast period, growth is expected due to increased awareness and diagnosis, expanded access to treatments, higher healthcare investments in rare diseases, a favorable regulatory environment, and increased healthcare expenditure. Key trends during this period include advancements in oral therapies, the adoption of gene therapy, technological innovations in drug delivery systems, the integration of artificial intelligence in SMA treatment development, and the use of gene editing technologies.

The increasing prevalence of rare genetic diseases is expected to drive the growth of the spinal muscular atrophy (SMA) market. Rare genetic disorders, caused by mutations in genes, affect a small segment of the population and often lead to severe, chronic health issues. This rise in rare genetic diseases can be attributed to advancements in diagnostic technologies, greater awareness, and improved access to genetic testing, which have resulted in the identification and reporting of more cases. Spinal muscular atrophy, a rare genetic disorder, serves as an example of the challenges and opportunities associated with understanding inherited diseases, highlighting the need for ongoing research in genetic conditions. For example, in July 2023, the Office for National Statistics in the UK reported that genetic testing of SMN1 has enabled more precise epidemiological studies, showing that SMA occurs in 1 in 10,000 to 20,000 live births and that over 95% of affected patients have homozygous SMN1 deletion. As a result, the rising prevalence of rare genetic diseases is expected to drive the growth of the SMA market.

Companies in the spinal muscular atrophy market are developing innovative treatments to improve medication adherence and ease of administration, particularly for pediatric patients and those with difficulty swallowing. One such innovation is dry syrup, a powdered medication that is reconstituted with water before use, often prescribed for children and patients who struggle to swallow pills or capsules. For instance, in September 2024, Chugai Pharmaceutical Co., Ltd., a pharmaceutical company based in Japan, received regulatory approval from Japan's Ministry of Health, Labour and Welfare for an expanded indication of its drug Evrysdi (risdiplam). This approval allows the drug's use in pre-symptomatic SMA and extends its dosage to infants under two months old. This approval is a significant milestone, as it allows treatment before the onset of symptoms, potentially improving therapeutic outcomes for affected infants. The decision was supported by data from the RAINBOWFISH study, which assessed the drug's safety and efficacy in infants diagnosed with SMA but not yet symptomatic. Results showed that treated infants reached developmental milestones, such as unsupported sitting and improved motor skills, by 12 months, demonstrating the drug's potential to alter the disease's progression when administered early.

In January 2024, Voyager Therapeutics, a US-based biotechnology company, entered into a partnership with Novartis AG to advance gene therapies for Huntington's disease and spinal muscular atrophy. The collaboration utilizes Voyager's proprietary TRACER capsids and advanced technology, while Novartis, a Swiss pharmaceutical company, brings its expertise in developing drugs and eye care products.

Major players in the spinal muscular atrophy market are Pfizer Inc., F. Hoffmann-La Roche Ltd, Bayer AG, Novartis AG, Biogen Inc., Chugai Pharmaceutical Co. Ltd., Genentech Inc., PTC Therapeutics Inc., Ionis Pharmaceuticals Inc., Biohaven Ltd., Catalyst Pharmaceuticals Inc., RegenXBio Inc., Cytokinetics Inc., AveXis Inc., Scholar Rock Inc., Genethon, CANbridge Pharmaceuticals Inc., NMD Pharma A/S, Hanugen Therapeutics Inc., and Beijing Jinlan Gene Technology Co. Ltd.

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

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder marked by the progressive degeneration of motor neurons in the spinal cord, leading to muscle weakness and atrophy. It is caused by mutations in the SMN1 gene, which results in a deficiency of survival motor neuron (SMN) protein. SMA primarily impacts voluntary muscle movements, particularly those necessary for walking, swallowing, and breathing.

SMA is categorized into four main types, type 1 (severe), type 2 (intermediate), type 3 (mild), and type 4 (adult). Type 1, or severe SMA, is the most critical form, characterized by profound muscle weakness and an inability to sit or move independently, often leading to death before the age of 2 without treatment. The condition affects both pediatric and adult patients. Treatment options include drug therapy and gene therapy, which are available through various end users, including hospital pharmacies, online pharmacies, and retail pharmacies.

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

The spinal muscular atrophy market consists of revenues earned by entities by providing services such as gene delivery services, diagnostic services, and supportive care services. The market value includes the value of related goods sold by the service provider or included within the service offering. The spinal muscular atrophy market also includes sales of therapy products, diagnostic kits, medical devices, and nutritional products. 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/or 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.

This product will be delivered within 3-5 business days.