Spinal Muscular Atrophy Market Size, Share, Growth, and Industry Analysis, By Type (Early Stage Candidates, Late Stage Candidates), By Application (Hospital, Clinic, Others), Regional Insights, and Forecast To 2031

Updated On: 29 April 2024

Spinal Muscular Atrophy Market Report Overview

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global spinal muscular atrophy market size was USD 1249.5 million in 2021 and market is projected to touch 3491.13 Million By 2031, exhibiting a CAGR of 10.7% during the forecast period.

Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy (wasting). It is primarily caused by mutations in the survival motor neuron 1 (SMN1) gene, which is responsible for the production of a protein called survival motor neuron (SMN). This protein is essential for the normal functioning of motor neurons, which control muscle movement.

SMA is typically inherited in an autosomal recessive manner, meaning that an individual must inherit a defective copy of the SMN1 gene from both parents to develop the condition. If a person has one defective gene and one normal gene, they are considered carriers and usually do not experience symptoms. The severity of SMA can vary widely, ranging from a severe form that begins in infancy and leads to significant disability and reduced life expectancy, to milder forms that manifest later in childhood or adulthood.

COVID-19 Impact

"Pandemic Hampered the Demand for Market"

The impact of COVID-19 on the Spinal Muscular Atrophy (SMA) market has been significant. While SMA itself is a rare genetic disorder, the pandemic has had broader implications for healthcare systems, research, and access to treatments, which have indirectly affected the SMA market.  Due to overwhelmed healthcare systems and diverted resources during the pandemic, there have been delays in the diagnosis and treatment of various conditions, including SMA. This has led to a potential increase in undiagnosed cases or delayed initiation of treatment, impacting the overall patient population and the demand for SMA therapies.  Many ongoing clinical trials for SMA and related therapies experienced disruptions due to COVID-19. Enrollment of new patients, monitoring visits, and follow-up assessments were affected, leading to delays in trial completion and regulatory approvals. These disruptions could have delayed the availability of new treatments or limited the data required for their approval.

Latest Trends

"Treatment Access and Affordability to Fuel Market Growth"

Access to spinal muscular atrophy treatments has been a significant concern due to their high costs. Efforts have been made to ensure broader access to these therapies through expanded insurance coverage, patient assistance programs, and negotiations on pricing. These initiatives aim to address the affordability issues associated with SMA treatments. Newborn screening programs have been expanding in various countries, enabling early detection of SMA. Early diagnosis allows for timely interventions and treatments, leading to better outcomes. Expanded screening programs aim to identify SMA cases at birth or in the pre-symptomatic stage, when interventions may be most effective. The approval of gene therapies has been a significant trend in the SMA market. Spinraza (nusinersen) was the first FDA-approved treatment for SMA, followed by Zolgensma (onasemnogene abeparvovec), which is a one-time gene replacement therapy. These treatments aim to address the underlying genetic cause of SMA and have shown promising results in improving motor function in patients.

Spinal Muscular Atrophy Market Segmentation

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• By Type Analysis

According to type, the market can be segmented into Early Stage Candidates, Late Stage Candidates.

• By Application Analysis

Based on application, the market can be divided into Hospital, Clinic, Others.

Driving Factors

"Regulatory Support to Stimulate Market Growth"

Regulatory agencies, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have played a vital role in expediting the development and approval of SMA treatments. Accelerated pathways, orphan drug designations, and priority reviews have supported the timely availability of novel therapies for SMA patients.  Collaborations between pharmaceutical companies, research institutions, and patient advocacy groups have accelerated progress in SMA research and development. Such collaborations facilitate knowledge sharing, resource pooling, and the efficient translation of scientific discoveries into effective therapies. Partnerships also play a crucial role in conducting clinical trials, expanding treatment access, and promoting patient advocacy.

"Several Advantages of Product to Fuel Market Growth "

The strong advocacy efforts of SMA patient organizations and support groups have significantly influenced the SMA market. These organizations raise awareness, provide support networks, and actively engage with stakeholders to drive research, treatment access, and policy changes. Patient stories and experiences have helped generate empathy and mobilize resources for SMA research and treatment development. All these factors are supporting spinal muscular atrophy market growth.

Restraining Factors

"High Cost of Treatments to Restrict Market Growth "

Spinal muscular atrophy treatments, such as gene therapies like Zolgensma and Spinraza, can be extremely expensive. The high costs associated with these therapies can pose a significant barrier to access for many patients, limiting their availability and affordability.  Due to the high costs of SMA treatments, reimbursement and insurance coverage can be limited, creating financial challenges for patients and their families. Insufficient coverage can restrict access to treatment options and impact the market growth.

Spinal Muscular Atrophy Market Regional Insights

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"Presence of Key Players in North America Anticipated to Drive Market Expansion"

North America holds leading position in spinal muscular atrophy market share. The United States has been at the forefront of spinal muscular atrophy research, diagnosis, and treatment, and it is home to several pharmaceutical companies actively involved in developing therapies for SMA. Furthermore, the U.S. Food and Drug Administration (FDA) approved the first-ever treatment for SMA called nusinersen (brand name Spinraza) in December 2016. Spinraza has been a significant advancement in the treatment of SMA and has contributed to the prominence of the U.S. in the market.

Key Industry Players

"Adoption Innovative Strategies by Key Players Influencing Market Growth"

Prominent market players are making collaborative efforts by partnering with other companies to stay ahead of the competition. Many companies are also investing in new product launches to expand their product portfolio.

The top key players in the market are Lonis Pharmaceuticals, F. Hoffmann-La Roche, AveXis, Novartis, Cytokinetics, Ionis Pharmaceuticals. The strategies to develop new technologies, capital investment in R&D, improve product quality, acquisitions, mergers, and compete for the market competition help them to perpetuate their position and value in the market. Besides, collaboration with other companies & extensive possession over market shares by the key players stimulates market demand.

List of Market Players Profiled

• Lonis Pharmaceuticals
• F. Hoffmann-La Roche
• AveXis
• Novartis
• Cytokinetics
• Ionis Pharmaceuticals

Report Coverage

This report examines an understanding of the spinal muscular atrophy market’s size, share, and growth rate, segmentation by type, application, key players, and previous and current market scenarios. The report also collects the market’s precise data and forecasts by market experts. Also, it describes the study of this industry’s financial performance, investments, growth, innovation marks, and new product launches by the top companies and offers deep insights into the current market structure, competitive analysis based on key players, key driving forces, and restraints that affect the demand for growth, opportunities, and risks.

Furthermore, the post-COVID-19 pandemic’s effects on international market restrictions and a deep understanding of how the industry will recover, and strategies are also stated in the report. The competitive landscape has also been examined in detail to provide clarification of the competitive landscape.

This report also discloses the research based on methodologies that define price trend analysis of target companies, collection of data, statistics, target competitors, import-export, information, and previous years’ records based on market sales. Moreover, all the significant factors which influence the market such as small or medium business industry, macro-economic indicators, value chain analysis, and demand-side dynamics, with all the major business players have been explained in detail. This analysis is subject to modification if the key players and feasible analysis of market dynamics change.