Spinal Muscular Atrophy: Forecast in Asia-Pacific Markets to 2028
- Pages: 104
- Published: November 2019
- Report Code: GDHC102POA
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.
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.
Allen & Hanburys
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?
3.2 Related Reports
4 Disease Overview
4.1 Etiology and Pathophysiology
4.2 Classification or Staging Systems
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
5.6.1 Epidemiological Forecast Insight
5.6.2 Limitations of Analysis
5.6.3 Strengths of Analysis
6 Current Treatment Options
7 Unmet Needs and Opportunity Assessment in APAC
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
8.1.1 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
9.2 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
10.4.4 South Korea
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
11.5 Primary Research – Prescriber Survey
11.6 About the Authors
11.6.2 Therapy Area Director
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
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