Global Spinal Muscular Atrophy Market
Healthcare Services

How is the Spinal Muscular Atrophy Market Poised for Growth: Trends and Opportunities Through 2034

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How has the spinal muscular atrophy market grown over the years?

In recent times, a swift expansion has been observed in the market size for spinal muscular atrophy. The growth projection indicates that it will increase from $3.53 billion in 2024 to hit $4.01 billion in 2025, marking the compound annual growth rate (CAGR) at 13.5%. Factors such as heightened diagnosis and early detection rates, a rising prevalence of rare genetic diseases, enhancements in healthcare facilities, support from governmental and regulatory bodies, as well as a boost in unresolved healthcare necessities, have all contributed to this substantial growth in the historical timeframe.

What Is the forecasted market size and growth rate for the spinal muscular atrophy market?

The market for spinal muscular atrophy is poised for significant expansion in the coming years, projecting to reach $6.59 billion by 2029, with a compound annual growth rate (CAGR) of 13.2%. The anticipated growth during the forecast period can be ascribed to factors such as increased awareness and diagnosis, improvement in treatment accessibility, a surge in healthcare investments towards rare diseases, a conducive regulatory environment, and escalating healthcare spending. Key trends expected to influence the market during the forecast period include progress in oral therapies, embrace of gene therapy, technological breakthroughs in drug delivery systems, the application of artificial intelligence in the development of SMA treatments, and emerging gene editing technologies.

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What are the major factors driving growth in the spinal muscular atrophy market?

The growth of the spinal muscular atrophy market is anticipated to be stimulated by the surging incidence of uncommon genetic diseases. Such conditions are the result of aberrations in the genes, affecting a small segment of the population and often leading to grave, persistent health issues. The escalating incidence can be ascribed to refined diagnostic tools, heightened awareness, and better access to genetic screenings, consequently identifying and reporting more cases. Spinal muscular atrophy (SMA), an unusual genetic condition, epitomizes the complexities and possibilities inherent in understanding hereditary diseases, and emphasizes the need for advancing research in the area of genetics. For example, a report issued by the Office for National Statistics, a UK governmental department, in July 2023 conveyed that SMN1 genetic testing has facilitated precise epidemiological studies. These studies illustrate that SMA affects 1 in every 10,000 to 20,000 live births, and over 95% of affected individuals are homozygous for SMN1 deletion. Consequently, the mounting incidence of rare genetic conditions will catalyze the expansion of the spinal muscular atrophy market.

What key areas define the segmentation of the global spinal muscular atrophy Market?

The spinal muscular atrophy market covered in this report is segmented –

1) By Type: Type 1 (Severe), Type 2 (Intermediate), Type 3 (Mild), Type 4 (Adult)

2) By Age: Pediatric, Adults

3) By Treatment: Drug Therapy, Gene Therapy

4) By End User: Hospital pharmacy, Online pharmacy, Retail pharmacy

Subsegments:

1) By Type 1 (Severe): Infantile-Onset SMA, Severe Progressive SMA

2) By Type 2 (Intermediate): Early-Onset SMA, Non-progressive SMA

3) By Type 3 (Mild): Juvenile-Onset SMA, Mild Progressive SMA

4) By Type 4 (Adult): Adult-Onset SMA, Late-Onset SMA

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What are the top market players propelling the growth of the spinal muscular atrophy industry?

Major companies operating 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., Beijing Jinlan Gene Technology Co. Ltd.

What key trends are currently impacting the spinal muscular atrophy market’s development?

Major companies operating in the spinal muscular atrophy market are developing innovative products, such as dry syrup, to improve medication adherence and ease of administration for pediatric and other patient groups with swallowing difficulties. Dry syrup is a powdered medication that is reconstituted with water before use, commonly prescribed for children and patients who have difficulty swallowing tablets or capsules. For instance, in September 2024, Chugai Pharmaceutical Co., Ltd., a Japan-based pharmaceutical company, announced regulatory approval from Japan’s Ministry of Health, Labour and Welfare for an expanded indication of its drug Evrysdi (risdiplam). The approval allows its use in pre-symptomatic spinal muscular atrophy (SMA) and extends dosing to infants under two months old. This milestone is crucial as it enables treatment initiation before symptoms appear, potentially enhancing therapeutic outcomes for affected infants. The decision was supported by data from the RAINBOWFISH study, which assessed Evrysdi’s safety and efficacy in infants diagnosed with SMA but not yet symptomatic. Findings showed that treated infants achieved developmental milestones, such as unsupported sitting and improved motor skills, by 12 months, underscoring the drug’s potential to transform the disease’s progression when administered early.

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What regions are dominating the spinal muscular atrophy market growth?

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 the spinal muscular atrophy market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

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