Spinal Muscular Atrophy (SMA) – Opportunity Analysis and Forecasts to 2028

SMA is an area that is expected to witness strong growth in the future, primarily due to launch of pipeline products that are expected to gradually capture market share from Biogen’s Spinraza. Also, the launch of survival motor neuron (SMN)- independent therapies are expected to be used in conjunction with an SMN-dependent therapy and this is also expected to contribute to SMA market growth in the forecast period.

Established pharmaceutical companies as well as smaller biotechnology companies are leading the development of pipeline products.

Developmental challenges include the need for long-term safety and clinical data for SMA drugs especially those that are proposed to work by gene-therapy, the need for early diagnosis of disease to ensure that the treatment has the maximum therapeutic effect, and the need to gain more understanding of the natural history of the disease by sharing data from previous trials and studies that may not have met the end-points but the results are capable of providing valuable insights on tried and tested methodologies in an effort to streamline various product development efforts.

Key Questions Answered

What are the key unmet needs in SMA? What do KOLs identify as the key unmet needs? Will the products in the current pipeline fill these unmet needs?

What were the key SMA treatments in 2018?

When will the late stage pipeline products launch, and how will it affect drug sales and the overall SMA market in the 7MM?


Overview of SMA, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and disease management.

Market Data including annualized SMA therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in fours patient segments (Type 1, 2, 3, and 4) forecast from 2018 to 2028.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the SMA therapeutics market.

Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for SMA therapy. The most promising candidates in Phase II and III development are profiled.

Analysis of the current and future market competition in the global SMA therapeutics market: Insightful review of the key industry drivers and barriers. Each trend is independently researched to provide qualitative analysis of its implications.

Key Highlights

The SMA market is expected to witness a CAGR of 16.0% from 2018 to 2028, reaching global value of $6.6 billion.

GlobalData anticipates that four pipeline drugs will launch over the forecast period in the US, the EU, and Japan. These pipeline drugs are expected to address significant unmet needs for SMA and significantly boost growth in the SMA market across the 7MM.

Among these pipeline products, two products are similar in its MOA as Biogen’s Spinraza which was the first approved therapy for SMA. Novartis’ Zolgensma, a gene therapy which received FDA approval in May 2019, is also anticipated to receive regulatory approvals in the EU and Japan. This therapy is proposed to be administered as a one-off infusion and is one that has excited all the KOLs interviewed by GlobalData. The aforementioned drugs are proposed to address the root cause of the disease. In addition to these, there is another set of drugs in pipeline development that are aimed at improving muscle functions in SMA patients.

While clinical unmet needs such as need for more treatment options are expected to be met by launch of pipeline products in the forecast period, environmental unmet needs such as market access barriers are expected to be a prominent topic of discussion among pharmaceutical companies and funding agencies and one that will evolve over the forecast period to help develop sustainable funding mechanisms for expensive drugs.

Reasons to buy

The report will enable you to:

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

Develop business strategies by understanding the trends shaping and driving the global SMA therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global SMA therapeutics market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Companies mentioned

Astellas Pharma


Catalyst Pharmaceuticals


Kowa Co Ltd


PTC Therapeutics


Scholar Rock

Table of Contents

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Spinal Muscular Atrophy: Executive Summary

2.1 Launch of Pipeline Products as Well as Newborn Screening Initiatives Are Expected to Drive the SMA Market Forward

2.2 Distinct R&D Strategies Help Companies to Tackle SMA and Reap Benefits

2.3 Environmental Unmet Needs Outweigh Clinical Unmet Needs

2.4 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

3.3 Upcoming Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Classification of Spinal Muscular Atrophy

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.4.3 Forecast Assumptions and Methods – Diagnosed Prevalent Cases of SMA

5.5 Epidemiological Forecast for SMA (2018–2028)

5.5.1 Diagnosed Prevalent Cases of SMA

5.5.2 Sex-Specific Diagnosed Prevalent Cases of SMA

5.5.3 Diagnosed Prevalent Cases of SMA by Type

5.6 Discussion

5.6.1 Epidemiological Forecast Insight

5.6.2 Limitations of Analysis

5.6.3 Strengths of Analysis

6 Current Treatment Options

6.1 Overview

7 Unmet Needs and Opportunity Assessment

7.1 Overview

7.2 Novel Mechanisms of Action and Favorable Routes of Administration

7.3 Early Diagnosis of Disease

7.4 Need to Understand the Natural History of Disease

7.5 Removal of Access Barriers

8 R&D Strategies

8.1 Overview

8.1.1 Development of a Novel Gene Therapy that Addresses the Cause of 5q SMA and Requires One-Time Administration

8.1.2 Development of Oral Agents that can Modulate RNA Splicing

8.1.3 Development of Therapeutic Agents that Can Increase Strength and Muscle Function

8.2 Clinical Trials Design

8.2.1 Outcome Measures and Trial Designs

8.2.2 Patient Population, Recruitment, and Retention

8.2.3 Trial Duration

9 Pipeline Assessment

9.1 Overview

9.2 Innovative Early-Stage Approaches

9.3 Other Drugs in Development

10 Pipeline Valuation Analysis

10.1 Clinical Benchmark of Key Pipeline Drugs

10.2 Commercial Benchmark of Key Pipeline Drugs

10.3 Competitive Assessment

10.4 Top-Line 10-Year Forecast

10.4.1 US

10.4.2 5EU

10.4.3 Japan

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

11.3.1 Forecasting Methodology

11.3.2 Diagnosed Prevalent Cases of SMA Patients

11.3.3 Percent Drug-Treated Patients

11.3.4 Drugs Included in Each Therapeutic Class

11.3.5 Launch and Patent Expiry Dates

11.3.6 General Pricing Assumptions

11.3.7 Individual Drug Assumptions

11.3.8 Generic Erosion

11.3.9 Pricing of Pipeline Agents

11.4 Primary Research – KOLs Interviewed for This Report

11.4.1 KOLs

11.4.2 Payers

11.5 Primary Research – Prescriber Survey

11.6 About the Authors

11.6.1 Analyst

11.6.2 Therapy Area Director

11.6.3 Epidemiologist

11.6.4 Reviewers

11.6.5 Global Director of Therapy Analysis and Epidemiology

11.6.6 Global Head and EVP of Healthcare Operations and Strategy

11.7 About GlobalData

11.8 Contact Us

11.9 Disclaimer

List of Tables

Table 1: Spinal Muscular Atrophy: Key Metrics in the Seven Markets

Table 2: Clinical Classification of SMA

Table 3: Risk Factors and Comorbidities for SMA

Table 4: Treatment Guidelines for Spinal Muscular Atrophy

Table 5: Leading Treatments for Spinal Muscular Atrophy, 2019

Table 6: Comparison of Therapeutic Classes in Development for SMA, 2018–2028

Table 7: Innovative Early-Stage Approaches for SMA, 2019

Table 8: Drugs in Development for SMA, 2019

Table 9: Clinical Benchmark of Key Pipeline Drugs – SMA

Table 10: Commercial Benchmark of Key Pipeline Drugs – SMA

Table 11: Key Events Impacting Global Sales for Spinal Muscular Atrophy, 2018–2028

Table 12: Spinal Muscular Atrophy Market – Global Drivers and Barriers, 2018–2028

Table 13: Key Historical and Projected Launch Dates for SMA

Table 14: Key Historical and Projected Patent Expiry Dates for SMA

Table 15: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

Figure 1: Global Sales Forecast by Country for Spinal Muscular Atrophy in 2018 and 2028

Figure 2: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Spinraza

Figure 3: Difference Between SMN1 and SMN2 Genes and The Molecular Basis of SMA

Figure 4: Diagnostic Evaluation for Spinal Muscular Atrophy

Figure 5: 7MM, Diagnosed Prevalence of SMA, %, Both Sexes, All Ages, 2018

Figure 6: 7MM, Sources Used and Not Used to Forecast the Diagnosed Prevalent Cases of SMA

Figure 7: 7MM, Diagnosed Prevalent Cases of SMA, N, Both Sexes, All Ages, 2018

Figure 8: 7MM, Sex-Specific Diagnosed Prevalent Cases of SMA, N, All Ages, 2018

Figure 9: 7MM, Diagnosed Prevalent Cases of SMA by Type, N, Both Sexes, All Ages, 2018

Figure 10: Unmet Needs and Opportunities in Spinal Muscular Atrophy

Figure 11: Overview of the Development Pipeline in SMA

Figure 12: Key Trials for the Promising Pipeline Agents that GlobalData Expects to Be Licensed for SMA During the Forecast Period

Figure 13: Competitive Assessment of the Marketed and Pipeline Drugs Benchmarked Against the SOC, Spinraza

Figure 14: Global Sales Forecast by Country for SMA in 2018 and 2028


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