Spinal Muscular Atrophy – Forecast in Asia-Pacific Markets to 2028

Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disease that primarily affects children. It is characterized by loss of spinal motor neurons, which are specialized cells that control muscle movement.

In recent years the first approved therapy for SMA, Biogen’s Spinraza (nusinersen), has gained marketing authorization in four of the five growth markets (5GM) – Japan, Australia, South Korea and China.

Strong market growth is anticipated across the 5GM over the forecast period. This will largely be driven by increased uptake of Spinraza in the early-to-mid forecast period and the launch of pipeline products from 2020 onwards.

Key unmet environmental and clinical needs in SMA include limited SMA resources and specialists, lack of disease awareness among physicians, low diagnosis rates, limited treatment options and lack of access to disease-modifying therapy for late-onset SMA.

Key Questions Answered

How is SMA currently managed and does this vary by subtype?

To what extent will the launch of pipeline products over the forecast period fulfil the unmet needs in the market?

How will the launch of pipeline products impact Spinraza?

How will growth vary by market?


Overview of SMA, including epidemiology, etiology, pathophysiology, classification, 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 unmet needs and opportunity assessment, R&D strategies and clinical trial design.

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 SMA therapeutics market: Insightful review of the industry drivers and barriers and in-depth analysis of trends within each of the 5GM.

Key Highlights

In the past three years, Spinraza, the first disease-modifying therapy for SMA has been approved in four of the 5GM – Japan, Australia, South Korea and China.

However, the market remains characterized by significant unmet needs, both clinical (e.g. limited treatment options) and environmental (e.g. lack of disease awareness among physicians).

GlobalData anticipates that four pipeline drugs will launch over the forecast period in the Asia-Pacific (APAC) region. These pipeline drugs are expected to address significant unmet needs for SMA and significantly boost growth in the SMA market across the 5GM.

These pipeline products include Novartis’ Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy administered as a one-time intravenous infusion, as well as risdiplam and branaplam, orally administered small molecules in development by Roche and Novartis, respectively, which have a similar mechanism of action to Spinraza.

Across the 5GM, the SMA market is projected to grow from $308.6M in 2018 to $2.5B in 2018 at a CAGR of 23.3%.

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 most active within the SMA APAC landscape.

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

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the SMA APAC 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

Allen & Hanburys





Kowa Co


PTC Therapeutics

Rhone-Poulenc Rorer



SMA Foundation

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 Uptake of New Products to Drive Robust Growth Within the SMA Market

2.2 Biogen to Lose its Monopoly of the SMA Market

2.3 Unmet Needs Remain High Despite Approval of First Disease-Modifying Therapy

2.4 Zolgensma and Risdiplam Demonstrate Greatest Potential in SMA

2.5 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

4.2 Classification or Staging Systems

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 Used

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 for Growth Markets (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 in APAC

7.1 Overview

7.2 Diagnostic Delays and Under-Diagnosis within the Patient Population

7.3 Poor Care and Lack of Access to Disease-Modifying Therapies for Late-Onset SMA

7.4 Novel Mechanisms of Action and Favorable Routes of Administration

7.5 Need to Understand the Natural History of the Disease

7.6 Lack of Social, Emotional, and Psychological Support for Patients and Carers

8 R&D Strategies in APAC

8.1 Overview

8.1.1 Development of a Novel Gene Therapy

8.1.2 Development of Oral Agents that can Modulate RNA Splicing

8.1.3 Development of a Generic Histone Deacetylase Inhibitor

8.1.4 Reliance on Strategic Consolidations

8.2 Clinical Trials Design

8.2.1 Patient Populations, Trial Size, and Trial Duration

8.2.2 Efficacy Endpoints Investigated

8.2.3 Comparator Arms

9 Pipeline Assessment in APAC

9.1 Overview

9.2 Promising Drugs in Clinical 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 India

10.4.2 China

10.4.3 Australia

10.4.4 South Korea

10.4.5 Japan

11 Appendix

11.1 Bibliography

11.2 Abbreviations

11.3 Methodology

11.3.1 Forecasting Methodology

11.3.2 Diagnosed 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 Pricing of Pipeline Agents

11.4 Primary Research – KOLs Interviewed for This Report

11.4.1 KOLs

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: SMA: Key Metrics in the 5GM in APAC

Table 2: Clinical Classification of SMA

Table 3: Risk Factors and Comorbidities for SMA

Table 4: Treatment Guidelines for SMA

Table 5: Leading Treatments for SMA, 2019

Table 6: Clinical Trials for Zolgensma (Onasemnogene Abeparvovec-xioi), 2019

Table 7: Clinical Trials for Risdiplam, 2019

Table 8: Clinical Trials for Valproate Sodium, 2019

Table 9: Comparison of Therapeutic Classes in Development for SMA, 2018–2028

Table 10: Clinical Benchmark of Key Pipeline Drugs – SMA

Table 11: Commercial Benchmark of Key Pipeline Drugs – SMA

Table 12: Key Events Impacting Sales for SMA, 2018–2028

Table 13: SMA Market – Global Drivers and Barriers, 2018–2028

Table 14: Key Historical and Projected Launch Dates for SMA

Table 15: Key Historical and Projected Patent Expiry Dates for SMA

Table 16: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

Figure 1: Global Sales Forecast by Country for SMA in 2018 and 2028

Figure 2: Competitive Assessment of the Late-Stage Pipeline Agents that GlobalData Expects to be Licensed for the Treatment of SMA During the Forecast Period

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

Figure 4: Diagnostic Evaluation for SMA

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

Figure 6: 5GM, Sources Used and Not Used to Forecast the Diagnosed Prevalent Cases of SMA

Figure 7: 5GM, Diagnosed Prevalent Cases of SMA, N, Both Sexes, All Ages, 2018

Figure 8: 5GM, Sex-Specific Diagnosed Prevalent Cases of SMA, N, All Ages, 2018

Figure 9: 5GM, Diagnosed Prevalent Cases of SMA by Type, N, Both Sexes, All Ages, 2018

Figure 10: Unmet Needs and Opportunities in SMA

Figure 11: Overview of the Development Pipeline SMA

Figure 12: Key Phase II/III Trials for the Promising Pipeline Agents that GlobalData Expects to be Licensed for SMA in the 5GM During the Forecast Period

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

Figure 14: 5GM Sales Forecast by Country for SMA in 2018 and 2028

Figure 15: Sales Forecast by Product for SMA in India in 2028

Figure 16: Sales Forecast by Product for SMA in China in 2018 and 2028

Figure 17: Sales Forecast by Product for SMA in Australia in 2018 and 2028

Figure 18: Sales Forecast by Product for SMA in South Korea in 2018 and 2028

Figure 19: Sales Forecast by Products for SMA in Japan in 2018 and 2028


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