Lysosomal Acid Lipase Deficiency Treatment Market

The global lysosomal acid lipase deficiency treatment market size is projected to expand at a significant CAGR during the forecast period, 2021–2028. The growth of the market is attributed to the increasing usage of enzyme replacement treatment, the ongoing use of supportive care therapies, and the increasing number of unmet requirements.

Lysosomal acid lipase deficiency (LAL-D) is a rare hereditary disease that is chronic and progressive. It impairs the body's capacity to generate a lysosomal acid lipase enzyme (LAL). In cells, this enzyme is required for the breakdown of fats (lipids) and cholesterol. Fats build in organs and tissues throughout the body when the LAL enzyme is absent or weak, causing liver dysfunction and elevated bad cholesterol, which is linked to cardiovascular disease. Fats (lipids) accumulate in dangerous levels in cells and tissues throughout the body in affected people, resulting in liver disease.

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There are two types of the condition. In the most severe and uncommon form, the disease develops in childhood. From childhood through late adulthood, the less severe version might appear. Within the first weeks of birth, lipids build throughout the body, notably in the liver, in the severe, early-onset type of lysosomal acid lipase deficiency. An enlarged liver and spleen (hepatosplenomegaly), inadequate weight growth, a yellow tint to the skin and whites of the eyes (jaundice), vomiting, diarrhea, fatty stool (steatorrhea), and poor nutritional absorption from meals are all symptoms of lipid buildup (malabsorption). Calcium deposits in small hormone-producing glands on top of each kidney (adrenal glands), low iron levels in the blood (anemia), and developmental delay are common in affected newborns. Scar tissue forms fast in the liver, resulting in liver disease (cirrhosis).

Multi-organ failure and severe malnutrition are common in infants with this kind of lysosomal acid lipase deficiency, and they seldom live past one year. Earlier, the two types of lysosomal acid lipase deficiency were considered to be distinct diseases. Wolman disease was the name of the early-onset form, and cholesteryl ester storage disease was the name of the later-onset type. These labels are still frequently used to distinguish between the types of lysosomal acid lipase deficiency, despite the fact that they both have the same genetic origin and are now considered forms of the same disease.

As many indications and symptoms of LAL-D are similar to those of other more prevalent illnesses including nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis, a diagnosis of the ailment can take months or even years (NASH). Patients might sometimes seem and feel well until the disease has progressed. A blood test that evaluates the activity of the LAL enzyme is used to diagnose LAL-D. Genetic testing and a liver biopsy are two more supporting procedures that a healthcare professional may conduct. Biopsy findings can assist identify liver damage, but they cannot be utilized to diagnose LAL-D because other illnesses can cause comparable liver damage.

Market Trends, Drivers, Restraints, and Opportunities

• Increasing use of enzyme replacement therapy for treatment is main factor driving the market during the forecast period.
• Increased prevalence of LAL deficiency-related liver complications is projected to boost the market growth during forecast period.
• High prevalence of lysosomal acid lipase deficiency and vast patient pool are anticipated to fuel the market growth during the forecast period.
• Initiatives by public and private institutes to spread awareness about the disorder is expected to propel the market growth in the coming years.
• High cost of treatment is projected to hamper the market during the forecast period.
• Lack of awareness LAL-D treatment presents as key challenge that can hinder the market growth during the forecast period.
• Increased R&D investments and technological advancements are expected to offer lucrative opportunities for the market players.

Scope of Lysosomal Acid Lipase Deficiency Treatment Market Report

The report on the global lysosomal acid lipase deficiency treatment market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.

Lysosomal Acid Lipase Deficiency Treatment Market Segment Insights

Cholesteryl ester storage disease segment is expected to account for a key share

Based on indication, the lysosomal acid lipase deficiency treatment market is bifurcated into Wolman disease (WD) and cholesteryl ester storage disease (CESD). The cholesteryl ester storage disease segment is expected to account for a key share of the market during the forecast period owing to a high prevalence of disease and a rising use of enzyme replacement treatment (ERT). CESD is more common in children and adolescents than WD and has a greater prevalence. Because of differences in patient phenotype, diagnosis is difficult, and many instances go unreported or misdiagnosed.

Wolman disease is a more severe type of the condition with an earlier onset. It arises as a result of the lack of activity of the LAL enzyme. Infants have no clinical symptoms at birth, but develop diarrhea, vomiting, jaundice, and enlarged livers and spleens within a few months. Furthermore, poor nutrition absorption in the small intestine causes individuals to lose a lot of weight.

Enzyme replacement therapy segment is expected to constitute a key share

Based on treatment class, the lysosomal acid lipase deficiency treatment market is segmented into enzyme replacement therapy, lipid-modifying agents (statins), liver transplant surgery, stem cell transplantation, and supportive care. The enzyme replacement therapy segment is expected to constitute a key share of the market during the forecast period due to high efficacy and long-term disease control. Low awareness and high treatment costs, on the other hand, remain key hurdles for the LAL-D therapy market, limiting penetration and creating a financial burden for patients in undeveloped and developing regions.

Supportive care and nutritional assistance are the mainstays of current LAL deficiency treatment. Lipid-lowering medications (statins) and a low-fat diet are used to control plasma lipid levels, apolipoprotein B production, and elevated cholesterol and triglycerides in patients. Furthermore, because of the disease's progressive nature, patients frequently require a hematopoietic stem cell transplant or a liver transplant. A liver transplant may provide relief from liver failure in LAL patients, but it does not address the underlying cause and may not prevent disease development in extra-hepatic organs such as the heart and kidneys. It is, nevertheless, still a viable choice for people who want to improve their odds of survival. Although hematopoietic stem cell transplantation has been performed in a few babies with lysosomal acid lipase (LAL) deficiency, it has been linked to problems, and long-term results are uncertain.

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North America is anticipated to dominate the market

On the basis of regions, the lysosomal acid lipase deficiency treatment market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. North America is anticipated to dominate the market during the forecast period. The regional market growth can be attributed to Increased Kanuma usage, high disease prevalence, and the existence of a large patient pool. Rapid novel innovations, drug development, and research are other factors that expected to fuel the market growth.

On the other hand, Europe is expected to exhibit a rapid growth rate in the coming years due to the availability of funds for rare disease research and the existence of a large patient population. Kanuma was approved for commercialization by the European Commission for the treatment of LAL-D patients. This is expected to contribute to the region's market growth.

Asia-Pacific is also expected to exhibit a rapid growth rate in the coming years due to the approval for Kanuma and improvement in disease diagnosis. Worldwide, there is an critical need for the development of a curative treatment strategy with enhanced efficacy, as well as the development of novel early diagnostic indicators to accelerate disease diagnosis.

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Segments

The global lysosomal acid lipase deficiency treatment market has been segmented on the basis of

Indication

• Wolman Disease (WD)
• Cholesteryl Ester Storage Disease (CESD)

Treatment Class

• Enzyme Replacement Therapy
• Lipid-Modifying Agents (Statins)
• Surgery (Liver Transplant)
• Stem Cell Transplantation
• Supportive Care

Regions

• Asia Pacific
• North America
• Latin America
• Europe
• Middle East & Africa

Key Players

• Alexion Pharmaceutical Inc.
• AstraZeneca plc.
• Merck & Co., Inc.
• Pfizer, Inc.
• Teva Pharmaceutical Industries Ltd.

Competitive Landscape

Key players competing in the lysosomal acid lipase deficiency treatment market are Alexion Pharmaceutical Inc., AstraZeneca plc., Merck & Co., Inc., Pfizer, Inc. and Teva Pharmaceutical Industries Ltd.

Some of the key business strategies employed by companies includes mergers, acquisitions, partnerships, collaborations, capacity expansion, and product launches to enhance their market shares.