Global Spinal Muscular Atrophy (SMA) Treatment Market Research Report: Forecast (2025-2030)

Market Definition

SMA is several related genetic neuromuscular diseases with proximal muscle weakness and atrophy. SMA is characterized by the selective loss of a group of nerve cells in your spinal cord known as lower motor neurons, or anterior horn cells. These co-ordinate muscle contractions or in other words, muscle activity. Without these neurons, muscles do not receive the nerve signals that make muscles contract or move.

Market Insights & Analysis: Global Spinal Muscular Atrophy (SMA) Treatment Market (2025-30):

The Global Spinal Muscular Atrophy (SMA) Treatment Market size was valued at around USD 2.3 billion in 2023 and is projected to reach around USD 30.2 billion by 2030. Along with this, the market is anticipated to grow at a CAGR of around 18.2% during the forecast period, i.e., 2025-30. It is projected that the progress in the Global Spinal Muscular Atrophy (SMA) Treatment Market will be propelled mainly by innovations in gene therapy in addition to the higher focus on research and development of rare genetic disorders. The growing awareness of SMA due to early identification is escalating the demand for SMA treatment.

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Moreover, the increasing governmental and healthcare organizations’ participation necessary to fund/subsidize these very expensive treatment programs is further growing the market and also helping in deploying new treatment options. For instance,

• August 2024: The Government of India has increased funding for rare diseases, especially Spinal Muscular Atrophy (SMA) allocating nearly USD 10 million this year.

Also, the increasing pipeline of drugs with promising potential, along with the addition of advanced technologies, including gene replacement therapies and antisense oligonucleotide (ASO) treatments, are enhancing the market growth. New disease-modifying therapies (DMTs) are emerging, and the evolving medical facilities in developing economies are opening new opportunities for the pharmaceutical industry for SMA treatment.

Global Spinal Muscular Atrophy (SMA) Treatment Market Driver:

Growing Incidence of the Disorder to Augment the Market – In terms of cost and healthcare services, the overall burden of spinal muscular atrophy is one of the highest in the rare disorder. With multiple incidences of spinal muscular atrophy popping up due to the increase in population, the requirement for SMA treatment is growing drastically on a global level. As per the Spinal Muscular Atrophy Foundation estimations, 1 in every 6000 to 10000 babies are diagnosed with this, despite being considered a rare disorder. Thus the rising rampancy of this disorder is one of the major factors that increases revenue generation from the market. Moreover, the rising awareness regarding novel therapies and the increasing number of product launches are some of the major factors deriving market growth. The high need for quality treatment options coupled with the awareness about the lethal backlash of the disorder will significantly aid in the development of the market.

Global Spinal Muscular Atrophy (SMA) Treatment Market Growth Opportunity:

Increasing Effects of Gene Replacement Therapy - The increasing effect of gene replacement therapy is creating future growth opportunities in the development of the Global Spinal Muscular Atrophy (SMA) Treatment Market. Single-course gene modification therapies including onasemnogene abeparvovec which directly address the genetic anomaly underlying SMA. It is a promising treatment for SMA patients with one-time treatment that would last for a lifetime. This is due to the medication particularly targeting the gene that is responsible for the SMA condition in the patient. This allows for an effective treatment of SMA patients. The development of this scenario will drive the market growth worldwide over the foreseeable future.

Global Spinal Muscular Atrophy (SMA) Treatment Market Challenge:

High Cost Associated with Advanced Therapies – The treatment and medication for Spinal Muscular Atrophy are very expensive. For instance, Zolgensma which is a gene therapy medication for Spinal Muscular Atrophy cost about USD 2.1 million during its single use. Another SMA medication called Spinraza which is administered four times a year throughout life costs around USD 350,000 for a single dosage. Companies attribute such high costs of the medications to huge investments in research and development to develop them and also they are the critical lifesaving drugs. The low affordability and health insurance companies shy away from providing coverage at such a huge price. Additionally, these expensive drugs do not guarantee to meet all the expectations of SMA patients. Thus, all these factors are hindering the growth & expansion of the SMA industry globally.

Global Spinal Muscular Atrophy (SMA) Treatment Market Trend:

Ongoing Innovations & Advancements in Gene Therapy – The innovation and developments are being done in the treatment and medications for Spinal Muscular Atrophy (SMA). This is critically important the develop medications that effectively cure the condition without causing larger side effects. The new research and development and innovation will also help lower the cost of these generally expensive medications.  Furthermore, newly FDA-approved gene therapies like Nusinersen (Spinraza) and Onasemnogene abeparvovec-xioi (Zolgensma) have shown promising results and are changing the paradigm of the SMA Treatment Market. These developments highlight the increasing innovation in gene therapy for SMA treatment will boost the market growth over the forecasted period.

Global Spinal Muscular Atrophy (SMA) Treatment Market (2025-30): Segmentation Analysis

The Global Spinal Muscular Atrophy (SMA) Treatment Market study of MarkNtel Advisors evaluates & highlights the major trends and influencing factors in each segment. It includes predictions for the period 2025–2030 at the global level. Based on the analysis, the market has been further classified as:

Based on Type:

• Type I
• Type II
• Type III
• Type IV

SMA Type I has the largest number of SMA patients accounting for about 60% of them worldwide, according to the National Organization for Rare Disorders. This is due to the disease getting detected early on during infancy. It is defined by extensive muscle stabilization and respiratory distress. Moreover, Type I SMAs are the most severe ones as the as typically the symptoms start to appear in the first six months of birth. This if not treated on time can limit the life expectancy of the infant to less than two years. Therefore, the segment has the largest market share due to life-threatening scenarios which makes its treatment crucially important. As the medical infrastructure in the world is improving the deployment of advanced detection and screening will identify the SMA early on during the infancy of the child. Hence the segment will continue to remain the most prominent one in the market over the foreseeable future.

Based on Treatment:

• Gene Therapy
• Disease-Modifying Drugs

In the Global Spinal Muscular Atrophy (SMA) Treatment Market gene therapy segment holds a market share of over 70%. This is primarily due to the fact that it can offer long and lasting viable solutions for patients' conditions while improving their outcomes dramatically. This is because gene therapies specifically target the gene that is responsible for the SMA. For example, the Food and Drug Administration (FDA) approved nusinersen (Spinraza) will get injected into the fluid present in the vicinity of the patient's spinal cord. This will target the specific gene that promotes the production of sensor motor neuron (SMN) protein, whose deficiency is responsible for SMA.

Global Spinal Muscular Atrophy (SMA) Treatment Market (2025-30): Regional Projections

Geographically, the Global Spinal Muscular Atrophy (SMA) Treatment Market expands across:

• North America
• South America
• Europe
• The Middle East & Africa
• Asia-Pacific

North America is dominating the Global Spinal Muscular Atrophy Market accounting for more than 40% market share in 2024. The presence of the United States (USA) in the region fuels market growth due to the presence of well-developed medical research and development facilities, technology, and infrastructure. This leads to the development and deployment of innovative therapies and medications that would be critical to diagnosing and treating SMAs. This has led to a significant number of patients seeking treatment for SMA to look for it in the country.  Furthermore, the USA has an early detection and screening process to detect SMAs during childbirth and infancy which identifies the SMA patients and diagnoses them for further treatment. In addition, North American countries feature a vibrant pharmaceutical industry, with manufacturers, care organizations, and research institutions cooperating closely, augmenting SMA treatment delivery.

Global Spinal Muscular Atrophy (SMA) Treatment Industry Recent Development:

•  October 2024: Biogen Inc. reported data concerning the efficacy of nusinersen under the higher dose regimen from Parts B and C of the DEVOTE combined Phase 2/3 trial for infantile-onset, as well as later-onset spinal muscular atrophy in pre-treated-and-untreated-patients.
• March 2023, innovation in Zolgensma®, female name onasemnogene abeparvovec, was claimed by Novartis as a fitting one-time gene therapy essential for spinal muscular atrophy.

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