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

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Published Date : Jun 2019

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No. of Pages : 88 Pages

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 Biogens 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 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 Biogens 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.

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.

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.
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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