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Global Lysosomal Acid Lipase Deficiency Treatment Market Size, Trends & Analysis - Forecasts to 2026 By Treatment (Enzyme Replacement Therapy, Lipid-Modifying Agents (Statins), Surgery (Liver Transplant), and Supportive Care), By Indication (Wolman Disease (WD) and Cholesteryl Ester Storage Disease (CESD)), By Region (North America, Asia Pacific, CSA, Europe, and the Middle East and Africa); End-User, Landscape, Company Market Share Analysis, and Competitor Analysis
Lysosomal acid lipase deficiency is a disease related to metabolic lipid storage. It is a genetically acquired condition described by issues including the breakdown & utilization of fats & cholesterol in the body. For people affected with this disease, a relatively high content of fat (lipids) collects in the cells and tissues of the body, which ordinarily causes liver illness. There are two types of lysosomal acid lipase deficiency conditions. The most extreme start in the early stages, while the less extreme conditions can start from youth until middle age. In the case of an extreme deficiency, just after the baby is born, the early-onset form of accumulation process of lipids begins to grow in the body, with the liver being the worst-hit area, leading to a number of abnormalities such as an enlarged liver, excessive weight loss, yellowish skin, vomiting, diarrhea and inefficiency in absorbing the nutrients out of the food.
Lysosomal acid lipase deficiency is very rare and occurs in only 1 out of 500,000 individuals (also varies with respect to the population of the region), mostly newborn babies. The Food and Drug Administration's (FDA) authorization of novel drugs is likely to boost commercial demand for lysosomal acid lipase deficiency treatment. For instance, Kanuma (sebelipase alfa), an advanced enzyme replacement therapeutic (ERT) developed by Alexion Pharmaceutical, was certified by the FDA in 2015.
As per the National Institute for Health and Care Excellence (NICE) 2019, about 70% of individuals with lysosomal acid lipase deficiencies have a risk of developing liver problems and requiring a liver transplant. The rising prevalence rates of lysosomal acid lipase deficiency-related liver problems are projected to drive up the requirement for liver transplants, eventually accelerating the expansion in the lysosomal acid lipase deficient treatment market throughout the forecast period. The primary restriction for the development of the lysosomal acid lipase deficiency treatment market is the significant price and absence of knowledge regarding management and therapy amongst the population. The constraint that is acting to be a potential restraint to the market is the extremely high treatment cost of the disease.
The factors driving the growth of this market are the improved healthcare services globally and recently developed medical institutions dedicated especially to research in the field of children’s disease to find efficient and cost-effective therapeutics for the treatment of lysosomal acid lipase in the core demographics. Additionally, the increasing number of pediatric hospitals across the globe to provide the appropriate medical care and treatment to an infant right from the beginning of the disease is helping to boost the overall demand in the lysosomal acid lipase deficiency treatment market.
Globally, governments have been investing huge amounts of money to make leading organizations, including both profit and non-profit organizations, solely focus on children’s care and development. Moreover, biologists and scientists have been doing extensive research to find the cure for various genetic mutation diseases including lysosomal acid lipase deficiency. Bio-Pharmaceutical companies are working on coming up with new and better drugs which can be even more cost-effective so that they can be affordable for the core demographics of both developed and developing economies. Thus, these aforementioned drivers will lead to the growth in the global markets of lysosomal acid lipase deficiency treatment.
Based on treatment, lysosomal acid lipase deficiency treatment can be segregated into enzyme replacement therapy, lipid-modifying agents (statins), surgery (liver transplant), and supportive care. Enzyme replacement therapy (ERT) is the leading segment as per treatment outlook owing to the high usage of Sebelipase Alfa, also known as Kanuma globally. The segment's dominance is attributed to its features, namely, better survival rate and efficiency. It is also the recommended medication since it is the sole licensed and FDA-approved medication for this disorder.
Based on indication, lysosomal acid lipase deficiency treatment can be segregated into Wolman disease (WD) and cholesteryl ester storage disease (CESD). Wolman disease (WD) is leading the indication segment as it gets diagnosed in the very early stage of birth. As a result, the treatment starts immediately, hence driving the medication and drug market at a rapid rate. Wolman syndrome is an extraordinarily rare condition that affects both men and women equally. Thus, leading to the supremacy of this segment globally.
As per the geographical analysis, the market for lysosomal acid lipase deficiency market can be classified into North America (the US, Canada, and Mexico), Asia Pacific (India, China, Japan, Malaysia, Singapore, and Rest of Asia Pacific), Europe (Germany, United Kingdom, Italy, France, Spain, Netherlands, and Rest of Europe), Middle East & Africa and Central & South America. North America is assumed to predominate the lysosomal acid lipase (LAL) deficiency treatment market owing to accelerated innovative advances, drug production, and testing. Moreover, the region’s supremacy is attributed to the increasing number of children getting affected by Wolman Disease (WD). Also, the U.S. is investing extensively in research and development projects for the introduction of novel medicines and treatment processes for various diseases which currently have no permanent cure. The FDA has been acting proactively for drug approvals in the United States. Many notable companies are obtaining approvals for novel medicines for infant-related diseases. The Asia Pacific is the fastest-growing market as it has been a leader in the development of its health care services sector and the emergence of various organizations dedicated to the treatment of infants whose families are unable to bear the high cost of treatment for lysosomal acid lipase deficiency. Furthermore, notable pharmaceutical companies in China, Japan, and India are growing rapidly since the Asia Pacific is the manufacturing hub, thereby increasing the business in the lysosomal acid lipase deficiency treatment market. Also, biogenetic companies are working in collaboration with pharma companies to find an effective cure for the treatment of lysosomal acid lipase deficiency, thereby decreasing the chances of morbidity in the region.
Alexion Pharmaceutical Inc., AstraZeneca Plc., Merck & Co. Inc., Pfizer Inc., Teva Pharmaceutical Industries Ltd., LUPIN, Lannett, Hetero Healthcare Limited, Zydus Cadila, Sandoz International GmbH, BioCrick BioTech, Actiza Pharmaceutical Private Limited, among others, are the top players in the lysosomal acid lipase deficiency treatment market.
Please note: This is not an exhaustive list of companies profiled in the report.
Chapter 1 Methodology
1.1 Market Scope & Definitions
1.2 Estimates & Forecast Calculation
1.3 Historical Data Overview And Validation
1.4 Data Sources
1.4.1 Secondary
1.4.2 Primary
Chapter 2 Report Outlook
2.1 Global Lysosomal Acid Lipase Deficiency Treatment Market Overview, 2016-2026
2.1.1 Industry Overview
2.1.2 Treatment Overview
2.1.3 Indication Overview
2.1.4 Regional Overview
Chapter 3 Global Lysosomal Acid Lipase Deficiency Treatment Market Trends
3.1 Market Segmentation
3.2 Industry Background, 2016-2026
3.3 Market Key Trends
3.3.1 Positive Trends
3.3.1.1 Increasing number of morbidities related to new-born
babies
3.3.1.2 Medical institutions dedicated specially to the research
in the field of children’s disease
3.3.2 Industry Challenges
3.3.2.1 Extremely high treatment cost of the disease
3.4 Prospective Growth Scenario
3.4.1 Treatment Growth Scenario
3.4.2 Indication Growth Scenario
3.5 COVID-19 Influence over Industry Growth
3.6 Porter’s Analysis
3.7 PESTEL Analysis
3.8 Value Chain & Supply Chain Analysis
3.9 Regulatory Framework
3.9.1 North America
3.9.2 Europe
3.9.3 APAC
3.9.4 LATAM
3.9.5 MEA
3.10 Technology Overview
3.11 Market Share Analysis, 2020
3.11.1 Company Positioning Overview, 2020
Chapter 4 Global Lysosomal Acid Lipase Deficiency Treatment Market, By Treatment
4.2 Enzyme Replacement Therapy
4.2.1 Market Size, By Region, 2016-2026 (USD Million)
4.3 Lipid-Modifying Agents (Statins)
4.3.1 Market Size, By Region, 2016-2026 (USD Million)
4.4 Surgery (Liver Transplant)
4.4.1 Market Size, By Region, 2016-2026 (USD Million)
4.5 Supportive Care
4.5.1 Market Size, By Region, 2016-2026 (USD Million)
Chapter 5 Global Lysosomal Acid Lipase Deficiency Treatment Market, By Indication
5.1 Indication Outlook
5.2 Wolman Disease (WD)
5.2.1 Market Size, By Region, 2016-2026 (USD Million)
5.3 Cholesteryl Ester Storage Disease (CESD)
5.3.1 Market Size, By Region, 2016-2026 (USD Million)
Chapter 6 Global Lysosomal Acid Lipase Deficiency Treatment Market, By Region
6.1 Regional outlook
6.2 North America
6.2.1 Market Size, By Country 2016-2026 (USD Million)
6.2.2 Market Size, By Treatment, 2016-2026 (USD Million)
6.2.3 Market Size, By Indication, 2016-2026 (USD Million)
6.2.4 U.S.
6.2.4.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.2.4.2 Market Size, By Indication, 2016-2026 (USD Million)
6.2.5 Canada
6.2.5.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.2.5.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3 Europe
6.3.1 Market Size, By Country 2016-2026 (USD Million)
6.3.2 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.3 Market Size, By Indication, 2016-2026 (USD Million)
6.3.4 Germany
6.3.4.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.4.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3.5 UK
6.3.5.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.5.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3.6 France
6.3.6.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.6.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3.7 Italy
6.3.7.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.7.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3.8 Spain
6.3.8.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.8.2 Market Size, By Indication, 2016-2026 (USD Million)
6.3.9 Russia
6.3.9.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.3.9.2 Market Size, By Indication, 2016-2026 (USD Million)
6.4 Asia Pacific
6.4.1 Market Size, By Country 2016-2026 (USD Million)
6.4.2 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.3 Market Size, By Indication, 2016-2026 (USD Million)
6.4.4 China
6.4.4.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.4.2 Market Size, By Indication, 2016-2026 (USD Million)
6.4.5 India
6.4.5.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.5.2 Market Size, By Indication, 2016-2026 (USD Million)
6.4.6 Japan
6.4.6.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.6.2 Market Size, By Indication, 2016-2026 (USD Million)
6.4.7 Australia
6.4.7.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.7.2 Market size, By Indication, 2016-2026 (USD Million)
6.4.8 South Korea
6.4.8.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.4.8.2 Market Size, By Indication, 2016-2026 (USD Million)
6.5 Latin America
6.5.1 Market Size, By Country 2016-2026 (USD Million)
6.5.2 Market Size, By Treatment, 2016-2026 (USD Million)
6.5.3 Market Size, By Indication, 2016-2026 (USD Million)
6.5.4 Brazil
6.5.4.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.5.4.2 Market Size, By Indication, 2016-2026 (USD Million)
6.5.5 Mexico
6.5.5.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.5.5.2 Market Size, By Indication, 2016-2026 (USD Million)
6.5.6 Argentina
6.5.6.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.5.6.2 Market Size, By Indication, 2016-2026 (USD Million)
6.6 MEA
6.6.1 Market Size, By Country 2016-2026 (USD Million)
6.6.2 Market Size, By Treatment, 2016-2026 (USD Million)
6.6.3 Market Size, By Indication, 2016-2026 (USD Million)
6.6.4 Saudi Arabia
6.6.4.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.6.4.2 Market Size, By Indication, 2016-2026 (USD Million)
6.6.5 UAE
6.6.5.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.6.5.2 Market Size, By Indication, 2016-2026 (USD Million)
6.6.6 South Africa
6.6.6.1 Market Size, By Treatment, 2016-2026 (USD Million)
6.6.6.2 Market Size, By Indication, 2016-2026 (USD Million)
Chapter 7 Company Landscape
7.1 Competitive Analysis, 2020
7.2 Alexion Pharmaceutical Inc.
7.2.1 Company Overview
7.2.2 Financial Analysis
7.2.3 Strategic Positioning
7.2.4 Info Graphic Analysis
7.3 AstraZeneca Plc.
7.3.1 Company Overview
7.3.2 Financial Analysis
7.3.3 Strategic Positioning
7.3.4 Info Graphic Analysis
7.4 Merck & Co. Inc.
7.4.1 Company Overview
7.4.2 Financial Analysis
7.4.3 Strategic Positioning
7.4.4 Info Graphic Analysis
7.5 Pfizer Inc.
7.5.1 Company Overview
7.5.2 Financial Analysis
7.5.3 Strategic Positioning
7.5.4 Info Graphic Analysis
7.6 Teva Pharmaceutical Industries Ltd.
7.6.1 Company Overview
7.6.2 Financial Analysis
7.6.3 Strategic Positioning
7.6.4 Info Graphic Analysis
7.7 LUPIN
7.7.1 Company Overview
7.7.2 Financial Analysis
7.7.3 Strategic Positioning
7.7.4 Info Graphic Analysis
7.8 Lannett
7.8.1 Company Overview
7.8.2 Financial Analysis
7.8.3 Strategic Positioning
7.8.4 Info Graphic Analysis
7.9 Hetero Healthcare Limited
7.9.1 Company Overview
7.9.2 Financial Analysis
7.9.3 Strategic Positioning
7.9.4 Info Graphic Analysis
7.10 Zydus Cadila
7.10.1 Company Overview
7.10.2 Financial Analysis
7.10.3 Strategic Positioning
7.10.4 Info Graphic Analysis
7.11 Sandoz International GmbH
7.11.1 Company Overview
7.11.2 Financial Analysis
7.11.3 Strategic Positioning
7.11.4 Info Graphic Analysis
7.12 BioCrick BioTech
7.12.1 Company Overview
7.12.2 Financial Analysis
7.12.3 Strategic Positioning
7.12.4 Info Graphic Analysis
7.13 Actiza Pharmaceutical Private Limited
7.13.1 Company Overview
7.13.2 Financial Analysis
7.13.3 Strategic Positioning
7.13.4 Info Graphic Analysis
7.14 Other Companies
7.14.1 Company Overview
7.14.2 Financial Analysis
7.14.3 Strategic Positioning
7.14.4 Info Graphic Analysis
The Global Lysosomal Acid Lipase Deficiency Treatment Market has been studied from the year 2019 till 2026. However, the CAGR provided in the report is from the year 2021 to 2026. The research methodology involved three stages: Desk research, Primary research, and Analysis & Output from the entire research process.
The desk research involved a robust background study which meant referring to paid and unpaid databases to understand the market dynamics; mapping contracts from press releases; identifying the key players in the market, studying their product portfolio, competition level, annual reports/SEC filings & investor presentations; and learning the demand and supply-side analysis for the Lysosomal Acid Lipase Deficiency Treatment Market.
The primary research activity included telephonic conversations with more than 50 tier 1 industry consultants, distributors, and end-use product manufacturers.
Finally, based on the above thorough research process, an in-depth analysis was carried out considering the following aspects: market attractiveness, current & future market trends, market share analysis, SWOT analysis of the company and customer analytics.
This FREE sample includes market data points, ranging from trend analyses to market estimates & forecasts. See for yourself.
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