The spinal muscular atrophy treatment market is estimated to grow from US$4.824 billion in 2022 to US$7.716 billion in 2029 at 6.94% (CAGR). Spinal muscular atrophy is a disorder that affects the motor neurons, thereby causing them to lose overall control over voluntary muscular muscle movement. Approximately 1 in 10,000 persons have a hereditary condition known as spinal muscular atrophy (SMA).
The majority of SMA-reporting cases are caused by a missing SMN1 gene, which is necessary for producing SMN protein that improves muscular movements. The severity of SMA varies in degrees and can lead to early death if proper treatment and therapy are not given. Children suffering from spinal muscular atrophy have limited mobility and experience breathlessness, eating & swallowing problems, and muscle weakness.
Spinal Muscular Atrophy Treatment Market Drivers:
New genetic discoveries in gene therapy and therapeutic approaches, followed by the growing public awareness regarding spinal muscular atrophy, have provided a new path for market growth. Patient activism, newborn screening programs, and regulatory backing have all contributed to accelerating SMA therapy advancements. With more research being conducted and more accessible therapy alternatives, there is increasing hope for better results and a higher standard of living for those with SMA.
Additionally, numerous methods are used to treat spinal muscular atrophy. These include antisense oligonucleotide treatment with Spinraza, which aims to increase the synthesis of SMN proteins, and gene replacement therapies like Zolgensma, which provides a functioning SMN1 gene. Evrysdi and other small-molecule medications also target the development of SMN proteins. Besides raising the level of SMN protein in the body, emphasis is laid on the peripheral and central nervous systems. These methods are frequently referred to as non-SMN methods. Numerous non-SMN methods concentrate on the nerves or muscles.
Favorable R&D projects aimed at treating Spinal Muscular Atrophy offer a significant growth driver for the spinal muscular atrophy treatment market growth. These projects, which range from gene therapy to small molecule medications and gene editing technologies, are driven by the urgent need for efficient treatments and a better understanding of the genetic causes of SMA.
For instance, in October 2023, Genentech presented positive results from its RAINBOWFISH study, which analyzed the safety and efficacy of the “Evrysdi” drug in babies suffering from pre-symptomatic SMA.
Moreover, several R&D pipelines are currently in the clinical stage, which gives hope for a full recovery from SMA. For instance, in March 2024, Novartis presented new results from its study analyzing the clinical benefits of one-time gene therapy “Zolgensma” for the treatment of SMA in children. Furthermore, with current R&D projects, including DEVOTE, RESPOND, and ASCEND, Biogen hopes to evaluate treatment alternatives and provide guidance to the SMA community as per their choice.
Neurological illness has become a major concern, and to prevent its prevalence in adults and children, various strategic investments in new research followed by collaborations are being implemented, such as NeuroNext (Network for Excellence in Neuroscience Clinical Trials) formed by the National Institute of Neurological Disorders and Stroke (NINDS). The network's goal is to create early-stage studies that evaluate novel treatments and uncover biomarkers, which are often physical characteristics or substances found in physiological fluids like blood that can be used to analyze the existence of SMA disease in the body.
One of these projects was finding biomarkers for SMA and comprehending the underlying mechanisms and causes of the illness. Information from this research was used to determine whether to approve businesses (SpinrazaTM).
In screening programs, state public health laboratories utilize dried blood spots taken from a baby's heel and run a test to see if the missing gene sequence that causes SMA is the source of the disorder. Hence, if the SMN1 gene is found to be abnormal, further investigation is done to determine the number of copies of the SMN2 gene.
The serious impact left by spinal muscular atrophy in the human body has raised concerns, and to tackle such an issue, the addition of SMA screening of babies in national screening programs is being implemented. For instance, in November 2023, Ireland’s health ministry added spinal muscle atrophy to the country’s “National Newborn Blood Screening Program”, which would screen babies aged three to five days for the early detection of such neurological conditions.
Spinal Muscular Atrophy Treatment Market Geographical Outlook
The spinal muscular atrophy treatment market, region-wise, is analyzed into North America, South America, Europe, the Middle East and Africa, and Asia Pacific, where the latter is expected to show steady growth and will account for a considerable market share. The advent of novel therapeutics, including gene replacement therapies and small molecule pharmaceuticals, as well as increased awareness and the deployment of newborn screening programs, are assisting in expanding the treatment scale for patients.
Additionally, various schemes and initiatives, such as the Indian government’s “National Policy of Rare Disease”, have been implemented to bolster the treatment of rare diseases. These include neurological disorders such as spinal muscle atrophy, which have further contributed to the regional market expansion.
Spinal Muscular Atrophy Treatment Market Players and Products:
Spinal Muscular Atrophy Treatment Market Key Developments
| Report Metric | Details |
|---|---|
| Study Period | 2021 to 2031 |
| Historical Data | 2021 to 2024 |
| Base Year | 2025 |
| Forecast Period | 2026 – 2031 |
| Report Metric | Details |
| Growth Rate | CAGR of 12.35% from 2022 to 2029 |
| Study Period |
2019 to 2029 |
| Historical Data |
2019 to 2022 |
| Base Year | 2023 |
| Forecast Period | 2024 – 2029 |
| Forecast Unit (Value) | USD Billion |
| Segments Covered |
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| Companies Covered |
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| Regions Covered | North America, South America, Europe, Middle East and Africa, Asia Pacific |
| Customization Scope | Free report customization with purchase |
The Spinal Muscular Atrophy Treatment market is segmented and analyzed as: