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Published: Oct, 2018 | Pages:
65 | Publisher: Grand View Research
Industry: Pharmaceutical | Report Format: Electronic (PDF)
The global lysosomal acid lipase deficiency (LAL-D) treatment market size is likely to reach USD 954 million by 2025, as per a new report by Grand View Research, Inc., exhibiting a 10.8% CAGR during the forecast period. High unmet needs, rising prevalence, and increasing uptake of enzyme replacement therapy are some of the primary factors driving the market. LAL deficiency (LAL-D) is a rare type of lysosomal storage disorder in which patients are unable to breakdown cholesteryl esters and triglycerides due to a mutation in the LAL gene. As a result, there is an accumulation of lipids in the liver, spleen, and walls of blood vessels. LAL deficiency is a progressive disease with multi-systemic clinical manifestations, significant medical complications, and early mortality due to liver failure, especially among infants. Until 2015, the global LAL deficiency treatment market witnessed modest growth due to lack of approved treatments for the disease. Medical care was limited to supportive care, with surgery and lipid-modifying drugs such as statins remained the mainstay for treatment. In 2015, approval of Alexion Pharma’s Kanuma (sebelipase alfa) marked entry of enzyme replacement therapy (ERT) in the LAL-D space. Kanuma is a hydrolytic lysosomal cholesterol ester and triacylglycerol specific enzyme, administered intravenously in patients with LAL deficiency. Currently, Kanuma is the only drug approved for both Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD) in the key markets. Based on treatment, supportive care was the leading revenue contributor in the market in 2017. However, the ERT segment is expected to register the fastest growth during the forecast period, supported by continued uptake of Kanuma. Disease prognosis remains poor, as a majority of patients are either undiagnosed or misdiagnosed. Thus, there remains a strong need for extensive research to understand disease biology and develop effective therapeutic options with goal to overcome resistance and minimize toxicity. Further key findings from the report suggest: • CESD manifests later in life and is more prevalent among Caucasians and Hispanic populations, affecting approximately 1 in 300,000 individuals • Germany is at the forefront of the Europe market and is driven by high disease prevalence and availability of drugs in the ERT class • Approval of Kanuma in ERT class highlight a fundamental shift in treatment approach to LAL-D deficiency • The ERT segment is estimated to be the fastest growing during the forecast period, owing to Kanuma’s first-mover advantage as the only long-term treatment in untapped LAL-D patient population pool • Statins are anticipated to lose market share due to rising competition from generics within the class and increasing uptake of Alexion’s Kanuma • Rising eligible patient population and limited therapeutic intervention for LAL deficiency offer strong commercial opportunity to players to invest in developing novel treatments in this market • Lack of awareness in disease diagnosis and delayed access to medications due to poor prognosis may restrain the growth of the market
Table of Contents
Chapter 1 Report Scope
1.1 Indication Scope
1.2 Treatment Scope
1.3 Country Scope
1.4 Estimates and Forecast Timeline
Chapter 2 Methodology
2.1 Research Methodology
2.1.1 Information procurement
2.2 Information or Data Analysis
2.3 Market Formulation & Validation
2.4 List of Sources
2.5 List of Abbreviations
Chapter 3 Executive Summary
Chapter 4 Disease Primer and Epidemiology
4.1 Disease Primer
4.1.1 Stages and symptoms
4.1.2 Types
4.1.2.1 Wolman Disease
4.1.2.2 Cholesteryl Ester Storage Disease
4.1.3 Causes
4.1.4 Symptoms
4.2 Epidemiology
4.2.1 EPIDEMIOLOGICAL Forecast FOR LAL - D FOR Seven Major Markets (U.S., Japan, EU5)
Chapter 5 Global LAL - D Treatment Market Overview
5.1 Introduction and Market Overview
5.2 Market, by Treatment
5.2.1 Enzyme Replacement Therapy
5.2.2 Lipid Modifying Agents (Statins)
5.2.3 Surgery
5.2.4 Supportive Care
5.3 Market, by Indication
5.3.1 Wolman Disease
5.3.2 Cholesteryl Ester Storage Disease
5.4 Market Size and Forecast by Country (2017 - 2025)
5.4.1 Sales Performance by geographic markets, in $ million, 2017 - 2025
5.4.2 U.S.
5.4.3 Japan
5.4.4 U.K.
5.4.5 Germany
5.4.6 France
5.4.7 Spain
5.4.8 Italy
5.5 Market Share Distribution, by Company (2017 - 2025)
5.6 Market Dynamics and Brand Strategies
5.7 Patent Expiry Schedule
5.8 Lysosomal Acid Lipase Deficiency Market: Drivers and Restraints
5.8.1 Market Drivers and trends
5.8.2 Market Restraints
5.9 M & A, Deal Landscape (2014 - 2018 YTD)
5.9.1 Mergers & Acquistitions
5.9.2 Deals landscape
5.10 Pricing & Reimbursement
5.11 SWOT
Chapter 6 Company Profiles
6.1 Alexion Pharmaceuticals
6.1.1 Company Overview
6.1.2 Current Product Portfolio
6.1.3 Product Forecast Sales up to 2025
6.1.4 Strategic Initiatives
6.1.4.1 Key Company News Flow
6.1.4.2 Catalysts & events Calendar
6.1.5 SWOT
Chapter 7 Market Outlook
7.1 Winners and Losers
7.1.1 Winners
7.1.2 Losers
7.2 Emerging Companies/ New Technology Platforms
7.3 What the Future Holds
List of Tables Table 1 List of Abbreviation Table 2 Forecast Lysosomal Acid Lipase Deficiency (LAL - D) Prevalence, 2017 - 2025 Table 3 Forecast Lysosomal Acid Lipase Deficiency (LAL - D) Incidence, 2017 - 2025 Table 4 Sales by Indication, in $ Million, 2017 - 2025 Table 5 Sales by Treatment, in $ Million, 2017 - 2025 Table 6 Sales Performance by Geographic Markets, in $ Million Table 7 LAL - D Drugs Patent Expiry Schedule Table 8 Annual Cost Associated with LAL - D Treatment, by Country ($ Million) Table 9 Pricing of Currently Available Treatment for LAL - D Table 10 Alexion Pharmaceuticals Product Forecast Sales (2017 - 2025) Table 11 Product Profile: Kanuma (sebelipase alfa) Table 12 Alexion Pharma Product Forecast Sales (2017 - 2025) Table 13 Kanuma Phase III Study
List of Figures Fig. 1 Stages and Symptoms of Migraine Fig. 2 LAL - D Types Fig. 3 Global LAL - D Market Share, by Treatment (2017 - 2025) Fig. 4 Market Segmentation and Scope Fig. 5 LAL - D Market Share, by Indication (2017 - 2025) Fig. 6 LAL - D Treatment Market Share, by Country (2017 & 2025) Fig. 7 U.S. LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 8 Japan LAL - D Treatment Treatment Market, $ Million (2017 - 2025) Fig. 9 U.K. LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 10 Germany LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 11 France LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 12 Spain LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 13 Italy LAL - D Treatment Market, $ Million (2017 - 2025) Fig. 14 LAL - D Market Shares Distribution, by Company (2017 & 2025) Fig. 15 LAL - D Treatment Market Sector SWOT Fig. 16 Alexion Pharmaceuticals LAL - D Sales Forecast Fig. 17 Alexion Pharmaceuticals SWOT
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