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

The Spinal Muscular Atrophy Treatment Market is segmented by Type (Type I, Type II, Type III, and Type IV), Procedure (Gene Replacement Therapy and Drug Therapy), Route of Administration (Oral and Intrathecal), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The report offers the value (in USD million) for the above segments.

Market Snapshot

spinal muscular atrophy treatment market
Study Period: 2019-2027
Base Year: 2021
Fastest Growing Market: Asia Pacific
Largest Market: North America
CAGR: 14.5 %
spinal muscular atrophy therapeutics market

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Market Overview

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.

Scope of the Report

Spinal muscular atrophy linked to chromosome 5q (SMA) is a recessive, progressive, neuromuscular disorder caused by bi-allelic mutations in the SMN1 gene, resulting in motor neuron degeneration and variable presentation in relation to onset and severity. The spinal muscular atrophy treatment market is segmented by type (type I, type II, type III, and type IV), procedure (gene replacement therapy and drug therapy), route of administration (oral and intrathecal), and geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The report offers the value (in USD million) for the above segments. The report offers the value (in USD million) for the above segments.

By Type
Type 1
Type 2
Type 3
Type 4
By Procedure
Gene Replacement Therapy
Drug Therapy
Other Procedures
By Route of Administration
Oral
Intrathecal
By Geography
North America
United States
Canada
Mexico
Europe
Germany
United Kingdom
France
Italy
Spain
Rest of Europe
Asia-Pacific
China
Japan
India
Australia
South Korea
Rest of Asia-Pacific
Middle-East and Africa
GCC
South Africa
Rest of Middle-East and Africa
South America
Brazil
Argentina
Rest of South America

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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.

spinal muscular atrophy treatment market

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.

muscle atrophy/ wasting syndrome market

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.

Recent Developments

  • In August 2021, Novartis announced the lift of a partial clinical trial hold and planned to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with spinal muscular atrophy.
  • In July 2021, Roche launched prescription medicine Evrysdi in India, which is used for spinal muscular atrophy in adults and children aged two months and older.

Table of Contents

  1. 1. INTRODUCTION

    1. 1.1 Study Assumptions and Market Definition

    2. 1.2 Scope of the Study

  2. 2. RESEARCH METHODOLOGY

  3. 3. EXECUTIVE SUMMARY

  4. 4. MARKET DYNAMICS

    1. 4.1 Market Overview

    2. 4.2 Market Drivers

      1. 4.2.1 Growing Awareness Regarding Spinal Muscular Atrophy, Its Diagnostics, and Its Treatment

      2. 4.2.2 Rise in the Government Initiatives to Improve Healthcare Infrastructure

      3. 4.2.3 Increasing R&D Activities to Develop Novel Treatment Regimens for Rare Diseases

    3. 4.3 Market Restraints

      1. 4.3.1 High Cost of Treatment

      2. 4.3.2 Lack of Skilled Professionals for Surgical Procedures and Physiotherapy Regimens

    4. 4.4 Porter's Five Forces Analysis

      1. 4.4.1 Threat of New Entrants

      2. 4.4.2 Bargaining Power of Buyers/Consumers

      3. 4.4.3 Bargaining Power of Suppliers

      4. 4.4.4 Threat of Substitute Products

      5. 4.4.5 Intensity of Competitive Rivalry

  5. 5. MARKET SEGMENTATION (Market Size by Value – USD million)

    1. 5.1 By Type

      1. 5.1.1 Type 1

      2. 5.1.2 Type 2

      3. 5.1.3 Type 3

      4. 5.1.4 Type 4

    2. 5.2 By Procedure

      1. 5.2.1 Gene Replacement Therapy

      2. 5.2.2 Drug Therapy

      3. 5.2.3 Other Procedures

    3. 5.3 By Route of Administration

      1. 5.3.1 Oral

      2. 5.3.2 Intrathecal

    4. 5.4 By Geography

      1. 5.4.1 North America

        1. 5.4.1.1 United States

        2. 5.4.1.2 Canada

        3. 5.4.1.3 Mexico

      2. 5.4.2 Europe

        1. 5.4.2.1 Germany

        2. 5.4.2.2 United Kingdom

        3. 5.4.2.3 France

        4. 5.4.2.4 Italy

        5. 5.4.2.5 Spain

        6. 5.4.2.6 Rest of Europe

      3. 5.4.3 Asia-Pacific

        1. 5.4.3.1 China

        2. 5.4.3.2 Japan

        3. 5.4.3.3 India

        4. 5.4.3.4 Australia

        5. 5.4.3.5 South Korea

        6. 5.4.3.6 Rest of Asia-Pacific

      4. 5.4.4 Middle-East and Africa

        1. 5.4.4.1 GCC

        2. 5.4.4.2 South Africa

        3. 5.4.4.3 Rest of Middle-East and Africa

      5. 5.4.5 South America

        1. 5.4.5.1 Brazil

        2. 5.4.5.2 Argentina

        3. 5.4.5.3 Rest of South America

  6. 6. COMPETITIVE LANDSCAPE

    1. 6.1 Company Profiles

      1. 6.1.1 Novartis International AG

      2. 6.1.2 Biogen Inc.

      3. 6.1.3 Cytokinetics Inc.

      4. 6.1.4 F. Hoffmann - La Roche Ltd

      5. 6.1.5 Pfizer Inc.

      6. 6.1.6 Isis Pharmaceuticals Inc.

      7. 6.1.7 Sanofi Aventis Inc. (GenZyme Corporation)

      8. 6.1.8 Avexis Inc.

      9. 6.1.9 Catalyst Pharmaceutical

      10. 6.1.10 Chugai Pharmaceutical

    2. *List Not Exhaustive
  7. 7. MARKET OPPORTUNITIES AND FUTURE TRENDS

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Frequently Asked Questions

The Global Spinal Muscular Atrophy Treatment Market market is studied from 2019 - 2027.

The Global Spinal Muscular Atrophy Treatment Market is growing at a CAGR of 14.5% over the next 5 years.

Asia Pacific is growing at the highest CAGR over 2021- 2026.

North America holds highest share in 2021.

Biogen, F. Hoffmann - La Roche Ltd , Cytokinetics, Inc., , Novartis AG, Catalyst Pharmaceutical are the major companies operating in Global Spinal Muscular Atrophy Treatment Market .

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