The global spinal muscular atrophy treatment market was valued at $4.3 billion in 2022, and is projected to reach $8.4 billion by 2032, growing at a CAGR of 6.8% from 2023 to 2032.
Spinal muscular atrophy (SMA) is a genetic disorder that affects the motor neurons, which are the nerve cells responsible for controlling voluntary muscle movements. It is characterized by the progressive degeneration of these motor neurons, leading to muscle weakness and atrophy. The symptoms of SMA can vary widely, but they generally involve muscle weakness and atrophy, difficulty with motor function, respiratory problems, and reduced mobility. The severity of the condition and the rate of disease progression can also vary among individuals.
The most widely used and effective treatment for spinal muscular atrophy is gene replacement therapy. One such therapy is called Onasemnogene abeparvovec (Zolgensma). It involves delivering a functional copy of the SMN1 gene (which is deficient in SMA) to motor neurons using a viral vector. This therapy has shown remarkable success in infants with SMA and has the potential to halt disease progression.
The major factors that drive the growth of the global spinal muscular atrophy treatment market Include, Increase in prevalence of spinal muscular atrophy. For instance, according to the Centers for disease Control and Prevention (CDC) in March 2023, spinal muscular atrophy (SMA) is a genetic disorder that affects approximately 1 out of every 10,000 people.
As more cases are diagnosed and reported, there is a growing patient population that requires effective treatment options. This Increase in prevalence of SMA contributes to the expansion of the market growth. The introduction of disease-modifying therapies, such as Nusinersen and Onasemnogene abeparvovec, has revolutionized the treatment landscape for SMA. These therapies have shown significant clinical benefits and improved the prognosis for individuals with SMA, leading to Increased demand for SMA drugs and propels the market growth.
In addition, improved understanding, and awareness of SMA among healthcare professionals and the public, there has been an emphasis on early diagnosis through genetic testing. Early detection allows for timely intervention and treatment initiation, which is driving the demand for SMA therapies. Another market driver is the growing recognition of rare diseases significance, rise in initiatives to raise SMA awareness, and Increase in research efforts by market participants to develop effective treatments for SMA. However, high cost of the spinal muscular atrophy treatment limits the adoption of SMA drugs which restrain the market growth.
The global spinal muscular atrophy treatment market is segmented into type, route of administration, distribution channel, and region. By type, the market is categorized into type 1, type 2, and others. By route of administration, the market is bifurcated into oral and parenteral. By end user, the market is classified into hospitals and others. Region wise, the market is analyzed across North America (the U.S., Canada, and Mexico), Europe (Germany, France, the UK, Italy, Spain, and rest of Europe), Asia-Pacific (Japan, China, India, Australia, South Korea, and rest of Asia-Pacific), and LAMEA (Latin America and Middle East & Africa).
Major key players that operate in the global spinal muscular atrophy treatment market are Biogen, Beijing Jinlan Gene Technology Co., Ltd., Cytokinetics, F. Hoffmann-La Roche Ltd., Hanugen Theraputics, Ionis Pharmaceuticals, Inc. NMD PHARMA A/S, Novartis AG, Pfizer Inc., and Scholar Rock Holding Corporation. Key players operating in the market have adopted product approval, product launch, and agreement as their key strategies to expand their product portfolio.
Spinal muscular atrophy (SMA) is a genetic disorder that affects the motor neurons, which are the nerve cells responsible for controlling voluntary muscle movements. It is characterized by the progressive degeneration of these motor neurons, leading to muscle weakness and atrophy. The symptoms of SMA can vary widely, but they generally involve muscle weakness and atrophy, difficulty with motor function, respiratory problems, and reduced mobility. The severity of the condition and the rate of disease progression can also vary among individuals.
The most widely used and effective treatment for spinal muscular atrophy is gene replacement therapy. One such therapy is called Onasemnogene abeparvovec (Zolgensma). It involves delivering a functional copy of the SMN1 gene (which is deficient in SMA) to motor neurons using a viral vector. This therapy has shown remarkable success in infants with SMA and has the potential to halt disease progression.
The major factors that drive the growth of the global spinal muscular atrophy treatment market Include, Increase in prevalence of spinal muscular atrophy. For instance, according to the Centers for disease Control and Prevention (CDC) in March 2023, spinal muscular atrophy (SMA) is a genetic disorder that affects approximately 1 out of every 10,000 people.
As more cases are diagnosed and reported, there is a growing patient population that requires effective treatment options. This Increase in prevalence of SMA contributes to the expansion of the market growth. The introduction of disease-modifying therapies, such as Nusinersen and Onasemnogene abeparvovec, has revolutionized the treatment landscape for SMA. These therapies have shown significant clinical benefits and improved the prognosis for individuals with SMA, leading to Increased demand for SMA drugs and propels the market growth.
In addition, improved understanding, and awareness of SMA among healthcare professionals and the public, there has been an emphasis on early diagnosis through genetic testing. Early detection allows for timely intervention and treatment initiation, which is driving the demand for SMA therapies. Another market driver is the growing recognition of rare diseases significance, rise in initiatives to raise SMA awareness, and Increase in research efforts by market participants to develop effective treatments for SMA. However, high cost of the spinal muscular atrophy treatment limits the adoption of SMA drugs which restrain the market growth.
The global spinal muscular atrophy treatment market is segmented into type, route of administration, distribution channel, and region. By type, the market is categorized into type 1, type 2, and others. By route of administration, the market is bifurcated into oral and parenteral. By end user, the market is classified into hospitals and others. Region wise, the market is analyzed across North America (the U.S., Canada, and Mexico), Europe (Germany, France, the UK, Italy, Spain, and rest of Europe), Asia-Pacific (Japan, China, India, Australia, South Korea, and rest of Asia-Pacific), and LAMEA (Latin America and Middle East & Africa).
Major key players that operate in the global spinal muscular atrophy treatment market are Biogen, Beijing Jinlan Gene Technology Co., Ltd., Cytokinetics, F. Hoffmann-La Roche Ltd., Hanugen Theraputics, Ionis Pharmaceuticals, Inc. NMD PHARMA A/S, Novartis AG, Pfizer Inc., and Scholar Rock Holding Corporation. Key players operating in the market have adopted product approval, product launch, and agreement as their key strategies to expand their product portfolio.
Key Benefits For Stakeholders
- This report provides a quantitative analysis of the market segments, current trends, estimations, and dynamics of the spinal muscular atrophy treatment market analysis from 2022 to 2032 to identify the prevailing spinal muscular atrophy treatment market opportunities.
- The market research is offered along with information related to key drivers, restraints, and opportunities.
- Porter's five forces analysis highlights the potency of buyers and suppliers to enable stakeholders make profit-oriented business decisions and strengthen their supplier-buyer network.
- In-depth analysis of the spinal muscular atrophy treatment market segmentation assists to determine the prevailing market opportunities.
- Major countries in each region are mapped according to their revenue contribution to the global market.
- Market player positioning facilitates benchmarking and provides a clear understanding of the present position of the market players.
- The report Includes the analysis of the regional as well as global spinal muscular atrophy treatment market trends, key players, market segments, application areas, and market growth strategies.
Key Market Segments
By Type
- Type 1
- Type 2
- Others
By Route of Administration
- Oral
- Parenteral
By End User
- Hospitals
- Others
By Region
- North America
- U.S.
- Canada
- Mexico
- Europe
- Germany
- France
- UK
- Italy
- Spain
- Rest of Europe
- Asia-Pacific
- Japan
- China
- India
- Australia
- South Korea
- Rest of Asia-Pacific
- LAMEA
- Latin America
- Middle East And Africa
Key Market Players
- Beijing Jinlan Gene Technology Co., Ltd.
- Biogen
- Cytokinetics
- F. Hoffmann-La Roche Ltd.
- Hanugen Theraputics
- Ionis Pharmaceuticals, Inc.
- NMD Phrama A/S
- Novartis AG
- Pfizer Inc.
- Scholar Rock Holding Corporation
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Table of Contents
CHAPTER 1: INTRODUCTION
CHAPTER 2: EXECUTIVE SUMMARY
CHAPTER 3: MARKET OVERVIEW
CHAPTER 4: SPINAL MUSCULAR ATROPHY TREATMENT MARKET, BY TYPE
CHAPTER 5: SPINAL MUSCULAR ATROPHY TREATMENT MARKET, BY ROUTE OF ADMINISTRATION
CHAPTER 6: SPINAL MUSCULAR ATROPHY TREATMENT MARKET, BY END USER
CHAPTER 7: SPINAL MUSCULAR ATROPHY TREATMENT MARKET, BY REGION
CHAPTER 8: COMPETITIVE LANDSCAPE
CHAPTER 9: COMPANY PROFILES
List of Tables
List of Figures
Executive Summary
According to a new report, titled, 'Spinal Muscular Atrophy Treatment Market,' The spinal muscular atrophy treatment market was valued at $4.3 billion in 2022, and is estimated to reach $8.4 billion by 2032, growing at a CAGR of 6.8% from 2023 to 2032.The Spinal Muscular Atrophy Treatment Market is likely to experience a significant growth rate of 6.8% from 2023-2032 owing to increase in demand for spinal muscular drugs from pharmaceuticals sector.
Spinal muscular atrophy (SMA) encompasses a range of inherited disorders that can result in the degeneration and loss of specific nerve cells, known as motor neurons, in the brain and spinal cord. It is caused by a mutation in the survival motor neuron 1 (SMN1) gene, leading to a deficiency of SMN protein. SMA affects various types and ages of individuals, with SMA type 1 being the most severe and typically presenting in early infancy.
Key factors driving the growth of the spinal muscular atrophy treatment market include a rise in prevalence of spinal muscular atrophy, rise in government initiatives for spinal muscular atrophy (SMA), and rise in the demand for SMA drugs. SMA is a relatively common genetic disorder characterized by the loss of motor neurons in the spinal cord. Its prevalence and impact on patient lives highlighted the urgent need for effective treatments. For instance, according to the National Center for Biotechnology and Information (NCBI) in 2020, spinal muscular atrophy affected 1 in 6,000 to 10,000 live births caused by loss of the survival motor neuron 1 gene (SMN1). The rise in prevalence of the condition means that there is a significant demand for effective treatments to manage the symptoms and improve the quality of life for these patients.
In addition, ongoing R&D efforts lead to the introduction of new and more effective drugs to treat these conditions propels the market growth. Moreover, the increase in awareness among patients and healthcare professionals about spinal muscular atrophy and the importance of early diagnosis and treatment led to an increase in demand for spinal muscular atrophy treatment, further driving the growth of the market. Furthermore, rise in product approvals, availability of drugs such as Spinraza (nusinersen ), Zolgensma (onasemnogene abeparvovec), and Evrysdi (risdiplam) drive the market growth.
Furthermore, development of innovative therapies, such as gene replacement therapies and small molecule drugs, has provided new hope for SMA patients. The increase in demand for SMA drugs stems from the unmet medical need in the market, as they improve motor function, slow disease progression, and enhance quality of life.
The market also offers growth opportunities to the key players in the market. Increase in R&D activities in the field of spinal muscular atrophy (SMA) provides significant opportunities for market growth. Increased R&D efforts led to the development of new and improved treatment options for SMA. These include novel therapies, gene therapies, targeted drug delivery systems, and other innovative approaches. The introduction of more effective treatments expands the market by providing better solutions for patients with SMA. Thus, products under clinical evaluation are projected to provide lucrative opportunity for market growth.
The spinal muscular atrophy treatments market is segmented into type, route of administration, distribution channel, and region. On the basis of type, the market is categorized into type 1, type 2, and others. On the basis of the route of administration, it is categorized into oral and parenteral. On the basis of end user, it is fragmented into hospitals and others. Region wise, it is analyzed across North America (the U.S., Canada, and Mexico), Europe (the UK, Germany, France, Italy, Spain, and rest of Europe), Asia-Pacific (China, India, Japan, Australia, South Korea, and rest of Asia-Pacific), and LAMEA (Brazil, Saudi Arabia, South Africa, and rest of LAMEA).
The key players profiled in the study include Biogen, Beijing Jinlan Gene Technology Co., Ltd., Cytokinetics, F. Hoffmann-La Roche Ltd., Hanugen Theraputics, Ionis Pharmaceuticals, Inc. NMD PHARMA A/S, Novartis AG, Pfizer Inc., and Scholar Rock Holding Corporation. The players in the market have been actively engaged in the adoption of various strategies such as product approval, product launch, and agreement to remain competitive and gain an advantage over the competitors in the market. For instance, in April 2021, F. Hoffmann-La Roche Limited announced that Health Canada has granted EVRYSDI (risdiplam) market authorization for the treatment of spinal muscular atrophy (SMA) in patients two months of age or older.
Key Market Insights
- By type, the type 1 segment was the largest revenue contributor to the market and is estimated to reach $5,314.99 million by 2032, with a highest CAGR during the forecast period.
- By route of administration, the parenteral segment dominated the global market in 2022. However, the oral segment is expected to be the fastest-growing segment with a CAGR of 7.3% during the forecast period.
- Based on end user, the hospitals segment was the largest revenue contributor to the market in 2022. However, the other segment is expected to be the fastest-growing segment with a CAGR of 7.3% during the forecast period.
- Based on region, North America garnered the largest revenue share in 2022, whereas Asia-Pacific is anticipated to grow at the highest CAGR during the forecast period.
Companies Mentioned
- Beijing Jinlan Gene Technology Co., Ltd.
- Biogen
- Cytokinetics
- F. Hoffmann-La Roche Ltd.
- Hanugen Theraputics
- Ionis Pharmaceuticals, Inc.
- NMD Phrama A/S
- Novartis AG
- Pfizer Inc.
- Scholar Rock Holding Corporation
Methodology
The analyst offers exhaustive research and analysis based on a wide variety of factual inputs, which largely include interviews with industry participants, reliable statistics, and regional intelligence. The in-house industry experts play an instrumental role in designing analytic tools and models, tailored to the requirements of a particular industry segment. The primary research efforts include reaching out participants through mail, tele-conversations, referrals, professional networks, and face-to-face interactions.
They are also in professional corporate relations with various companies that allow them greater flexibility for reaching out to industry participants and commentators for interviews and discussions.
They also refer to a broad array of industry sources for their secondary research, which typically include; however, not limited to:
- Company SEC filings, annual reports, company websites, broker & financial reports, and investor presentations for competitive scenario and shape of the industry
- Scientific and technical writings for product information and related preemptions
- Regional government and statistical databases for macro analysis
- Authentic news articles and other related releases for market evaluation
- Internal and external proprietary databases, key market indicators, and relevant press releases for market estimates and forecast
Furthermore, the accuracy of the data will be analyzed and validated by conducting additional primaries with various industry experts and KOLs. They also provide robust post-sales support to clients.
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Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 239 |
| Published | June 2023 |
| Forecast Period | 2022 - 2032 |
| Estimated Market Value ( USD | $ 4.3 billion |
| Forecasted Market Value ( USD | $ 8.4 billion |
| Compound Annual Growth Rate | 6.9% |
| Regions Covered | Global |
| No. of Companies Mentioned | 10 |
