Spinal Muscular Atrophy Treatment Market – By Type (Werdnig-Hoffmann Disease, Kugelberg-Welander Disease), Treatment Type (Medication {Gene Therapy}, Supportive Care), Route of Administration (Oral, Parenteral), End-use – Global Forecast, 2024 ­ 2032

Spinal Muscular Atrophy Treatment Market Size

Spinal Muscular Atrophy Treatment Market size was valued at USD 7.3 billion in 2023 growing at a CAGR of 19.1% between 2024 and 2032. The key factors driving market growth include the expansion of newborn screening programs, which have led to earlier diagnoses of spinal muscular atrophy (SMA), increasing the number of patients eligible for treatment. Early diagnosis improves patient outcomes and boosts demand for medication to treat neurological conditions.
 

For instance, according to a report published by Health Union, LLC, SMA is one of the most common genetic conditions affecting children. One in every 6,000 to 10,000 babies worldwide is born with SMA. Type 1 SMA is the most severe form and accounts for more than half of all cases. Additionally, the prevalence of SMA is about 1 in 100,000 people globally. The prevalence is lower than the incidence due to the shorter lifespan associated with the most common SMA types. Approximately 25,000 Americans currently live with SMA. Thus, continuous advancements in diagnostic technology and increased awareness among healthcare providers and patients about SMA and its treatment options, leading to earlier interventions and higher treatment uptake, are anticipated to foster market growth.
 

Spinal muscular atrophy (SMA) treatment refers to medical interventions designed to manage and alleviate the symptoms of SMA, a genetic disorder characterized by the loss of motor neurons in the spinal cord, leading to muscle weakness and atrophy. Moreover, people with SMA often experience respiratory infections, scoliosis, and joint contractures (chronic shortening of muscles and tendons). While SMA primarily affects infants and children, it can also occur in adults.
 

Spinal Muscular Atrophy Treatment Market Trends

The ongoing innovation and advancement in gene therapy and other treatments for spinal muscular atrophy are anticipated to propel market growth during the analysis period. For instance, animal data presented in 2022 at the American Society of Gene and Cell Therapy (ASGCT), the European Society of Gene and Cell Therapy (ESGCT), and the World Muscle Congress showed that the gene therapy outperformed the benchmark therapy across multiple key endpoints in a mouse model of SMA and exhibited significantly less liver toxicity when administered intravenously. Thus, ongoing research into next-generation gene therapies and small molecule drugs is expanding the range of treatment options, attracting more investment and interest in the market, which is expected to contribute to its growth.
 

Spinal Muscular Atrophy Treatment Market Analysis

Based on the type, the market is classified into Werdnig-Hoffmann disease, infant SMA, Kugelberg-Welander disease, and adult SMA. The Werdnig-Hoffmann disease segment dominated the market with revenue of USD 2.9 billion in 2023.
 

• Werdnig-Hoffmann disease, also known as SMA Type 1, is the most severe form of spinal muscular atrophy and presents within the first six months of life. The implementation of mandatory newborn screening for SMA in many regions has led to earlier detection of Werdnig-Hoffmann disease. Early diagnosis is crucial, as it allows for the prompt initiation of treatment, which is most effective before significant motor neuron loss occurs.
   
• Moreover, ongoing research and development activities, coupled with an increase in partnerships between pharmaceutical companies aimed at reducing the cost of expensive SMA Type 1 treatments, have made these therapies more accessible to a broader patient population, boosting market growth.
   
• In addition, many pharmaceutical companies have implemented early access or compassionate use programs for SMA Type 1 treatments, allowing patients to receive therapies before formal approval. This helps build market momentum, thereby driving market growth.

Based on the treatment type, the spinal muscular atrophy treatment market is segmented into medication, supportive care, and spinal surgery. The medication segment is further bifurcated into gene therapy, antisense oligonucleotides, and other medications. The supportive care segment is classified into physical therapy, respiratory support, and nutritional support. The medication segment dominated the market with market share of 64.2% in 2023.
 

• The introduction of innovative drugs like Spinraza (Nusinersen) and Evrysdi (Risdiplam) has revolutionized the treatment of SMA. These medications specifically target the underlying genetic cause of the disease by increasing the production of survival motor neuron (SMN) protein, which is deficient in SMA patients, driving the growth of the market.
   
• In addition, the availability of both oral and injectable formulations has increased treatment accessibility, allowing patients with different preferences and needs to receive effective therapy.
   
• Thus, the flexibility in administration has broadened the appeal of SMA medications and contributed to their widespread adoption, thereby fueling market growth.
   

Based on the route of administration, the spinal muscular atrophy treatment market is segmented into oral and parenteral. The oral segment dominated the market and is expected to reach USD 19.5 billion by 2032.
 

• Oral SMA treatments can be administered at home without the need for clinical visits, reducing the burden on patients and their families. This convenience is particularly appealing for long-term treatment regimens, where frequent hospital visits can be disruptive, thereby driving market growth.
   
• In addition, oral therapies often reduce the need for healthcare resources, such as nursing support for injections or infusion centers, leading to cost savings for healthcare systems. These savings make oral treatments an attractive option for payers and providers, promoting their adoption and boosting market growth.
   

Based on end-use, the spinal muscular atrophy treatment market is classified into hospitals, specialty clinics, homecare settings, and other end-users. The hospitals segment is expected to exhibit 19.2% CAGR between 2024-2032.
 

• SMA treatment often involves a multidisciplinary approach, including neurologists, physical therapists, pulmonologists, and nutritionists. Hospitals are well-equipped to provide this coordinated care, ensuring that patients receive comprehensive treatment for their complex needs. This integrated approach enhances patient outcomes and contributes to the growth of the hospital segment.
   
• Moreover, hospitals often serve as sites for clinical trials of new SMA treatments, giving patients access to innovative therapies that may not be available elsewhere. This involvement in research enhances the hospital's reputation and attracts more patients seeking cutting-edge treatments, thereby fostering market growth.

In 2023, North America secured a substantial market share of 45.6% in the spinal muscular atrophy treatment market and is expected to dominate throughout the forecast period.
 

• North America, particularly the U.S., has implemented supportive policies for rare diseases, including SMA. These policies encourage innovation and provide incentives such as tax credits for research and development, further driving market growth.
   
• Moreover, the presence of some of the most advanced healthcare facilities and research institutions in North America allows for the widespread availability and adoption of cutting-edge SMA treatments like gene therapy and SMN-enhancing drugs.
   
• Furthermore, ongoing clinical trials in North America for new spinal muscular atrophy treatments are leading to an increased need for new treatments and the expansion of the therapeutic pipeline for SMA, which contributes to the growth of the market.
   

The spinal muscular atrophy treatment market in U.S. is expected to exhibit 19% CAGR between 2024 - 2032.
 

• Many states in the U.S. have implemented mandatory newborn screening for SMA, which has led to earlier diagnosis and intervention. Early detection significantly increases the likelihood of successful treatment, driving demand for SMA therapies.
   
• In addition, organizations like Cure SMA and the Muscular Dystrophy Association play a significant role in raising awareness, funding research, and advocating for better treatment options. These groups help educate patients, families, and healthcare providers about the latest SMA therapies, thereby boosting market growth.
   

Germany to experience the highest growth rate in the Europe market.
 

• Germany has a robust healthcare system with state-of-the-art medical facilities and highly skilled healthcare professionals. This infrastructure supports the delivery of advanced SMA treatments, including gene therapy and other innovative approaches, promoting market growth.
   
• Furthermore, Germany's regulatory body, the Federal Institute for Drugs and Medical Devices (BfArM), has streamlined the approval processes for innovative treatments, including orphan drugs for rare diseases like SMA. This has facilitated quicker access to new SMA therapies.
   

The Asia Pacific spinal muscular atrophy treatment market is expected to grow with a significant CAGR of 19.6% during the forecast period.
 

• There is increasing investment in R&D for SMA treatments within the Asia-Pacific region, driven by both local and global pharmaceutical companies. Collaborative research initiatives between local universities and global biotech firms are fostering innovation in SMA treatment options.
   
• Moreover, the availability and affordability of genetic testing have improved significantly in the region, allowing for more accurate diagnosis of SMA. Countries like China and India are witnessing a rise in genetic testing facilities, leading to better identification of SMA cases and a subsequent increase in treatment demand.
   

Spinal Muscular Atrophy Treatment Market Share

The market is characterized by intense competition among key players aiming to expand their market presence. Leading companies are focusing on strategic initiatives such as mergers, acquisitions, and partnerships to strengthen their market position. These efforts aim to enhance their product and service offerings, expand their geographic reach, and invest in advanced technologies. The market also witnesses the entry of new players, further intensifying the competitive landscape.
 

Spinal Muscular Atrophy Treatment Market Companies

The company profile section includes both companies that have commercial drug available in the market as well as those that are onto clinical phase development. Prominent players operating in the market are as mentioned below:

• American Physical Therapy Association
• Astellas Pharma
• Beijing Jinlan Gene Technology Co., Ltd.
• Biogen Inc.
• Boston's Children Hospital
• Children's Hospital of The King's Daughters
• F. Hoffmann-La Roche Ltd.
• Hanugen Therapeutics
• Nationwide Children’s Hospital
• NMD Pharma A/S
• Novartis AG
• Pfizer, Inc.
• Sanofi (Genzyme Corporation)
• Scholar Rock, Inc
   

Spinal Muscular Atrophy Treatment Industry News:

• In May 2022, Genentech, announced that the U.S. Food and Drug Administration (FDA) approved Evrysdi (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). Evrysdi is now approved to treat SMA in children and adults of all ages. This helped the company expand its product offerings.
   
• In March 2021, The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics, Inc. have entered a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders with the goal of developing new treatments. The SMA Foundation–PTC partnership will provide funding, managed by the SMA Foundation, to academic institutions and other collaborators to advance foundational research in regenerative medicine.
   

The spinal muscular atrophy treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2021 – 2032 for the following segments:

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Market, By Type

• Werdnig-Hoffmann disease
• Infant SMA
• Kugelberg-Welander disease
• Adult SMA

Market, By Treatment Type

• Medication
  • Gene therapy
  • Antisense oligonucleotides
  • Other medications
• Supportive care
  • Physical therapy
  • Respiratory support
  • Nutritional support
• Spinal surgery

Market, By Route of Administration

• Oral
• Parenteral

Market, By End-use

• Hospitals
• Specialty clinics
• Homecare settings
• Other end-users

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Italy
  • Spain
  • Netherlands
  • Rest of Europe
• Asia Pacific
  • Japan
  • China
  • India
  • Australia
  • South Korea
  • Rest of Asia Pacific
• Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America
• Middle East and Africa
  • South Africa
  • Saudia Arabia
  • UAE
  • Rest of Middle East and Africa