Spinal Muscular Atrophy Treatment Global Market Report 2025

The spinal muscular atrophy treatment market size has grown rapidly in recent years. It will grow from $4.41 billion in 2024 to $5.1 billion in 2025 at a compound annual growth rate (CAGR) of 15.7%. This expansion during the historical period was driven by a rising number of SMA cases, increased approvals of innovative gene therapies, heightened public awareness about early diagnosis and interventions, greater investment in R&D activities, and broader implementation of newborn screening initiatives.

The spinal muscular atrophy treatment market size is expected to see rapid growth in the next few years. It will grow to $9.02 billion in 2029 at a compound annual growth rate (CAGR) of 15.3%. Factors fueling this future growth include the ongoing rise in SMA prevalence, more approvals of cutting-edge gene therapies, amplified awareness surrounding early-stage treatment, continuous funding in scientific research, and an expanding reach of neonatal screening programs. Key trends expected in this period include improvements in gene therapy innovations, the adoption of personalized medicine techniques, the emergence of technology-enabled oral treatment options, progress in early diagnostic screening for newborns, and the development of combination therapy methodologies.

The increasing focus on gene therapy is expected to drive the growth of the spinal muscular atrophy (SMA) treatment market. Gene therapy involves modifying a person's genetic material, such as DNA or RNA, to treat or prevent diseases. This rising attention is largely due to gene therapy’s ability to address the root cause of genetic disorders by directly modifying or replacing defective genes, offering potentially long-term or permanent therapeutic benefits over traditional treatments. Treatments for spinal muscular atrophy, such as onasemnogene abeparvovec (Zolgensma), utilize gene therapy to provide a one-time, targeted solution that delivers a functional copy of the SMN1 gene. This approach significantly enhances motor function and quality of life for patients with SMA. For example, in January 2024, the American Society of Gene and Cell Therapy reported a 10% increase in the number of gene therapies in Phase III trials in the fourth quarter of 2023 compared to the previous quarter, marking the first such rise since the third quarter of 2022. This trend highlights how the growing emphasis on gene therapy is boosting the spinal muscular atrophy treatment market.

Leading companies in the spinal muscular atrophy treatment market are developing innovative formulations, such as survival motor neuron 2 (SMN2) splicing modifier tablets, to improve motor function and slow disease progression. These tablets are oral medications designed to modify the splicing of the SMN2 gene to promote the production of functional SMN protein, which is vital for motor neuron survival. By increasing the levels of full-length SMN protein, these treatments help improve muscle function and decelerate disease advancement. For instance, in February 2025, Genentech, Inc., a U.S.-based biotechnology company, received approval from the U.S. Food and Drug Administration for its Evrysdi (risdiplam) tablet. This tablet is the first and only non-invasive SMN2 splicing modifier available for treating SMA. Evrysdi offers similar efficacy and safety to the existing oral solution, along with additional benefits such as room-temperature storage and easier administration for patients aged two years and older who weigh more than 44 pounds (20 kilograms), enhancing convenience and flexibility in managing the disease.

In January 2024, Voyager Therapeutics Inc., a U.S.-based biotechnology firm, entered a partnership with Novartis to advance gene therapies for neurological diseases. This collaboration aims to develop novel gene therapies for conditions such as Huntington’s disease and spinal muscular atrophy by combining Voyager’s TRACER capsid technology with Novartis’s experience in gene therapy development and commercialization. Novartis, a pharmaceutical company headquartered in Switzerland, specializes in providing treatments for spinal muscular atrophy.

Major players in the spinal muscular atrophy treatment market are F. Hoffmann-La Roche Ltd, Novartis AG, Biogen Inc., Chugai Pharmaceutical Co. Ltd, PTC Therapeutics Inc., Ionis Pharmaceuticals Inc., Ultragenyx Pharmaceutical Inc., Catalyst Pharmaceuticals Inc., BIOCAD INDIA PRIVATE LIMITED, Cytokinetics Incorporated, Voyager Therapeutics Inc., Scholar Rock Holding Corporation, Genethon, Exegenesis Bio Inc, NMD Pharma A/S, Biohaven Pharmaceutical Holding Company Ltd, Alcyone Therapeutics Inc., Vybion Inc., Apteeus SAS, Biophytis SA, and Ractigen Therapeutics.

North America was the largest region in the spinal muscular atrophy treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in spinal muscular atrophy treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the spinal muscular atrophy treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Spinal muscular atrophy (SMA) treatment encompasses medical therapies and interventions aimed at enhancing muscle strength, improving motor abilities, and elevating the quality of life for individuals affected by SMA. This genetic disorder is marked by the progressive degeneration of motor neurons located in the spinal cord and lower brainstem.

The primary treatment options for spinal muscular atrophy include medications, physical therapy, surgical procedures, and other therapeutic approaches. Medications play a crucial role in alleviating symptoms, boosting motor performance, and slowing the progression of the disease. These therapies address various SMA types such as Werdnig-Hoffmann disease, infantile SMA, Kugelberg-Welander disease, and adult-onset SMA. Drug administration routes include oral and parenteral methods, and these medications are dispensed through hospital pharmacies, retail pharmacies, and other distribution networks. The key end users of these treatments include hospitals, homecare providers, specialty clinics, and others.

The spinal muscular atrophy treatment market research report is one of a series of new publications that offer comprehensive data on the spinal muscular atrophy treatment market, including global industry size, regional market shares, key players and their market positioning, detailed segmentation, current market trends, potential opportunities, and other essential insights. This report presents an all-encompassing view of the industry, offering a detailed examination of the present landscape and future prospects.

The spinal muscular atrophy treatment 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 treatment market also includes sales of spinraza, zolgensma, evrysdi, and supportive care 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.

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