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Spinal Muscular Atrophy Treatment Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)

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  • 110 Pages
  • August 2022
  • Region: Global
  • Mordor Intelligence
  • ID: 5012709
The spinal muscular atrophy treatment market is expected to register a CAGR of 14.5% during the forecast period.

Due to strict lockdown measures and restricted movement of people, the COVID-19 virus outbreak has had a significant impact on the spinal muscular atrophy medicine industry. The unprecedented coronavirus outbreak (SARS-CoV-2) has cast a shadow over the SMA treatment industry. For instance, according to the article published in the National Institute of Health in May 2020, titled "Spinal muscular atrophy care in the COVID‐19 pandemic era," the delay in the SMA treatment during the pandemic affected the delivery of clinical care to patients with spinal muscular atrophy (SMA). Moreover, the amount of money available for the R&D and drug development for SMA has been decreasing, as the majority of the money has been spent on COVID-19 vaccine production.

The major factors accounting for the growth of the spinal muscular atrophy treatment market are growing awareness regarding SMA, its diagnosis and treatment, rise in the government initiatives to improve healthcare infrastructure, and increasing R&D activities to develop novel treatment regimens. For instance, in August 2020, the US Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. This is the second drug and the first oral drug approved to treat this disease. Emerging treatment therapies in the treatment of SMA are driving the growth of the market during the forecast period.

However, the high cost of treatment and lack of skilled professionals for surgical procedures and physiotherapy regimens hampers the market to some extent.

Key Market Trends

The Gene Replacement Therapy Segment Dominates the Market, and the Same is Expected Over the Forecast Period

Gene replacement therapy is the technique of recognizing a faulty gene, applying a piece of DNA in its correct form through a viral vector (known as the carrier molecule) to the gene, thus, overriding the identified faulty gene with the correct copy.

Gene replacement therapy is expected to dominate the spinal muscular atrophy treatment market over the forecast period, attributing to its advantages over the disadvantages. However, investment in research and development activities is also expected to have a significant effect on the segment. Companies such as Pfizer Inc. are aiming to build a gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities and enhancing those capabilities through strategic collaborations, expansion of R&D activities, and potential licensing and merger and acquisition activities. For instance, in August 2019, Pfizer was investing USD 500 million to expand a manufacturing facility in North Carolina, which plays a central role in its efforts to become a major player in the gene therapy industry.

The effects of gene replacement therapy are long-lasting and improve the quality of life by eradicating genetic disorders. Recently in May 2019, the US FDA approved Zolgensma for the treatment of spinal muscular atrophy is a gene therapy, which is found to be the most efficient in results, and many other drugs are in the pipeline. Thus, gene replacement therapy dominates the spinal muscular atrophy treatment market. However, the healthcare cost for gene therapy using Zolgensma is high, but this may hamper the market to a very less extent, as the government is raising huge funds for the treatment of rare diseases. For example, under the government’s Ayushman Bharat scheme in India, the current draft policy provides up to INR 15 lakhs for rare disease treatment to patients who require one-time treatment.

Thus, all aforementioned factors, such as the advantages of gene replacement therapy and technological advancement in gene therapy, may boost the segment over the forecast period.

North America Dominates the Market, and the Same is Expected Over the Forecast Period

North America is expected to dominate the overall market throughout the forecast period, which is attributed to the developed economy, increasing health care expenditure, increasing incidence of lifestyle-related diseases, and high adoption of technologically advanced products in the region. According to the article published by Columbia University in November 2021, more than 7,000 rare diseases affect 25 million to 30 million American children and adults. More than 90% of rare diseases lack a Food and Drug Administration (FDA)-approved treatment. The reimbursement policies in the United States, especially for the treatment of rare diseases, further boost the market in North America.

Moreover, many key players in the market are focused on adopting strategies, such as mergers and acquisitions, to enhance their product portfolio, which, in turn, is expected to propel market growth over the forecast period. For instance, in August 2020, the US Food and Drug Administration approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Thus, North America dominates the spinal muscular atrophy treatment market during the forecast period.

Competitive Landscape

The spinal muscular atrophy treatment market is moderately competitive and consists of several major players. With the increasing consolidations of various organizations and product recalls in the healthcare sector, it is expected to generate competitive rivalry among the key players in the future. Some of the major players of the market are Novartis AG, Biogen Inc., F. Hoffmann - La Roche Ltd, and Cytokinetics Inc.

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Table of Contents

1.1 Study Assumptions and Market Definition
1.2 Scope of the Study
4.1 Market Overview
4.2 Market Drivers
4.2.1 Growing Awareness Regarding Spinal Muscular Atrophy, Its Diagnostics, and Its Treatment
4.2.2 Rise in the Government Initiatives to Improve Healthcare Infrastructure
4.2.3 Increasing R&D Activities to Develop Novel Treatment Regimens for Rare Diseases
4.3 Market Restraints
4.3.1 High Cost of Treatment
4.3.2 Lack of Skilled Professionals for Surgical Procedures and Physiotherapy Regimens
4.4 Porter's Five Forces Analysis
4.4.1 Threat of New Entrants
4.4.2 Bargaining Power of Buyers/Consumers
4.4.3 Bargaining Power of Suppliers
4.4.4 Threat of Substitute Products
4.4.5 Intensity of Competitive Rivalry
5 MARKET SEGMENTATION (Market Size by Value - USD million)
5.1 By Type
5.1.1 Type 1
5.1.2 Type 2
5.1.3 Type 3
5.1.4 Type 4
5.2 By Procedure
5.2.1 Gene Replacement Therapy
5.2.2 Drug Therapy
5.2.3 Other Procedures
5.3 By Route of Administration
5.3.1 Oral
5.3.2 Intrathecal
5.4 By Geography
5.4.1 North America United States Canada Mexico
5.4.2 Europe Germany United Kingdom France Italy Spain Rest of Europe
5.4.3 Asia-Pacific China Japan India Australia South Korea Rest of Asia-Pacific
5.4.4 Middle-East and Africa GCC South Africa Rest of Middle-East and Africa
5.4.5 South America Brazil Argentina Rest of South America
6.1 Company Profiles
6.1.1 Novartis International AG
6.1.2 Biogen Inc.
6.1.3 Cytokinetics Inc.
6.1.4 F. Hoffmann - La Roche Ltd
6.1.5 Pfizer Inc.
6.1.6 Isis Pharmaceuticals Inc.
6.1.7 Sanofi Aventis Inc. (GenZyme Corporation)
6.1.8 Avexis Inc.
6.1.9 Catalyst Pharmaceutical
6.1.10 Chugai Pharmaceutical

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

  • Novartis International AG
  • Biogen Inc.
  • Cytokinetics Inc.
  • F. Hoffmann - La Roche Ltd
  • Pfizer Inc.
  • Isis Pharmaceuticals Inc.
  • Sanofi Aventis Inc. (GenZyme Corporation)
  • Avexis Inc.
  • Catalyst Pharmaceutical
  • Chugai Pharmaceutical