Spinal Muscular Atrophy Treatment Market size was over USD 5.9 billion in 2024 and is anticipated to cross USD 53.02 billion by 2037, witnessing more than 18.4% CAGR during the forecast period i.e., between 2025-2037. In the year 2025, the industry size of spinal muscular atrophy treatment is estimated at USD 6.82 billion.
According to the National Library of Medicine, in October 2023, the incidence of SMA is estimated at 1 in 6000–10,000, with a carrier frequency of 1/40–1/60. Even though spinal muscular atrophy is considered a rare disease, the rising incidence of the cases, especially among newborn children is anticipated to boost the spinal muscular atrophy treatment market during the forecast period.
The growing prevalence of the genetic disorder has led to a rising awareness and demand for early diagnosis. Advances in gene therapies, and increasing investments in R&D, particularly in RNA-based therapies, have propelled the demand for novel, effective treatments. Governments and healthcare organizations are also playing a crucial role by providing incentives and fast-tracking drug approvals for rare diseases. Personalized medicine approaches, aim to improve patient outcomes by tailoring treatments to specific genetic profiles. Collaborations between biotech firms and academic institutions are further accelerating drug discovery, while patient advocacy groups are raising awareness and driving demand for innovative therapies in the spinal muscular atrophy treatment market.
Growth Drivers
Challenges
Treatment Type (Medication, Supportive Care, Spinal Surgery)
Based on treatment type, the medication segment is expected to register a considerable share in the spinal muscular atrophy treatment market. The segment is driven by the widespread use of RNA-based drugs such as Biogen’s Spinraza. Additionally, newer medicines including Evrysdi have provided more convenient and accessible options for patients, further bolstering the medication segment. These drugs are favored as they offer ongoing treatment options compared to one-time, high-cost gene therapies. Moreover, both oral and injectable formulations’ availability, improved accessibility to treatment, permitting different preferences and needs to be met with effective therapy. The continuous need for dosage over time provides a steady demand, making medications a key revenue-generating segment in the market.
Route of Administration (Oral, Parenteral)
Oral segment is likely to dominate spinal muscular atrophy treatment market share of over 62.9% by 2037. Administration of oral SMA treatments at home does not require clinical visits, reducing the load on patients and their families. It is particularly convenient for long-term treatment regimens, as visits to hospitals can be disruptive. This thereby drives the market growth significantly. Furthermore, oral therapies eliminate the need for healthcare resources including nursing support for injections or infusion centers. Cost savings due to this make oral treatments a more appealing option for payers and providers, hence, accelerating their adoption and boosting market growth.
Our in-depth analysis of the market includes the following segments:
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North America Market Analysis
Leading biotechnology firms, at the forefront of developing novel therapies, robust healthcare infrastructure, and strong government support are the leading factors driving the North America spinal muscular atrophy treatment market. The region has witnessed significant progress in terms of drug approvals and treatment availability. Advanced diagnostic capabilities, and established screening programs further fuel the growth of the SMA market in the region. Region’s reimbursement programs and insurance coverage options also help improve patient access across the region.
The U.S. spinal muscular atrophy treatment market is the largest globally, benefitting from the presence of key market players such as Biogen, and Novartis. The FDA’s supportive regulatory framework, including orphan drug designations, and fast-track approvals, has accelerated the development and commercialization of SMA therapies. For instance, in February 2023, Biohaven Ltd. received Fast Track designation from the U.S. FDA for taldefgrobep alfa which is a novel anti-myostatin adnectin, developed for the treatment of SMA.
Canada spinal muscular atrophy treatment market is supported by government-backed healthcare policies that provide access to expensive treatments through public health programs. Advancements in genetic testing and personalized medicine treatment are driving the market growth in Canada. Collaborations between research institutions and global biotech companies are also accelerating clinical trials and the development of innovative therapies in the country.
APAC Market Statistics
APAC spinal muscular atrophy treatment market is experiencing steady growth, driven by increasing awareness of SMA and improving healthcare infrastructure in countries such as India, Japan, Australia, and China. Government initiatives in the region are supporting the development and commercialization of SMA therapies. Furthermore, due to the region’s large population base, and growing focus on rare disease treatments, the Asia Pacific market is anticipated to register considerable growth.
India spinal muscular atrophy treatment market is in its early stages of development, with rising awareness and advocacy efforts to bring attention to the disease. However, the high cost of SMa treatments poses a barrier in the India market. The local government and healthcare organizations are making efforts to improve awareness, introduce affordable treatment options, and expand diagnostic capabilities. The country’s large population offers potential for future market growth as healthcare infrastructure improves.
Australia market is significantly more developed with established newborn screening programs, allowing early diagnosis and intervention. According to the country’s Department of Health and Aged Care, one in 35 Australians unknowingly carry the spinal muscular atrophy gene and one in 10,000 babies born in the country is affected by the disease. The government has been proactive in supporting rare disease treatments and offering reimbursement schemes such as the PBS. The country is hence anticipated to witness growth during the forecast period.
Companies in the spinal muscular atrophy treatment market are focusing on offering more effective long-term solutions. Many are investing in personalized medicines, targeting specific genetic mutations, while others are enhancing patient access through pricing strategies, reimbursement partnerships, and early screening programs. In October 2024, Roche presented two-year positive data from the ongoing study RAINBOWFISH at the 29th World Muscle Society assessing the efficacy and safety of risdiplam in children with SMA. Gaining regulatory approvals, and entering new markets are also a key strategy adopted by the prominent players.
Author Credits: Radhika Pawar
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