Product 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. The Spinal Muscular Atrophy (SMA) market across the five growth markets (5GM) Australia, China, India, Japan and South Korea in the Asia-Pacific (APAC) region is anticipated to grow at a compound annual growth rate of 23.3% from US$308.6m in 2018 to US$2.5bn in 2028. Increased uptake of the first disease-modifying Spinal Muscular Atrophy (SMA) therapies will drive the robust growth during the outlook period.
Biogen's antisense therapy Spinraza (nusinersen) was first approved for the treatment of Spinal Muscular Atrophy (SMA) in Japan in June 2017 and has since been approved in Australia, South Korea and China. The approval of this drug is revolutionizing the management of the disease, with clinically meaningful improvements in motor function, achievement of milestones and survival observed for the first time in children with Spinal Muscular Atrophy (SMA).
Spinraza accounted for the vast majority of sales across the (5GM) Australia, China, India, Japan and South Korea in 2018 and sales are projected to remain strong throughout the early-to-mid forecast period (2018-2023). However, Spinraza is projected to lose its monopoly in the mid-to-late forecast period (2023-2028), as pipeline products launching from 2020 onwards are expected to capture significant market share.
Key opinion leaders express excitement regarding the potential of the late-stage pipeline to expand access to therapy and address key unmet needs. In particular, Novartis' gene therapy Zolgensma (onasemnogene abeparvovec-xioi) represents a potentially transformative treatment option, as it is administered by a one-time intravenous infusion. This represents a strong differentiating factor from Spinraza, which requires intrathecal administration multiple times per year.
Zolgensma, which has already received approval in the US, is expected to emerge as the market leader in the APAC region by 2028 with sales of US$1.3bn across the (5GM) Australia, China, India, Japan and South Korea. Additionally, the launch of Roche's risdiplam, an SMN2 splicing modifier, is also eagerly anticipated owing to the drug's convenient oral administration route.
Although Spinraza's approval represented a major breakthrough in the field, there is still an unmet need for additional therapies that employ novel mechanisms of action and administration routes, which can translate into improvements in efficacy, accessibility, ease of use, and patient compliance.
The latest research report "Spinal Muscular Atrophy: Forecast in Asia-Pacific Markets to 2028" helps in answering the following question with regards to Spinal Muscular Atrophy (SMA) and its therapeutic market in Asia Pacific.
- How Spinal Muscular Atrophy (SMA) is currently managed and does this vary by subtype?
- To what extent will the launch of pipeline products over the forecast period fulfill 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.
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.
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?
- 3.1 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
- 5.1 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
7 Unmet Needs and Opportunity Assessment in APAC
8 R&D Strategies in APAC
9 Pipeline Assessment in APAC
- 9.1 Overview
- 9.2 Promising Drugs in Clinical Development
10 Pipeline Valuation Analysis
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