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Lysosomal acid lipase (LAL) deficiency is a rare inherited condition in which the body is not able to produce enough lysosomal acid lipase (LAL) enzyme, which is an important enzyme for breakdown of fatty materials namely cholesteryl esters and triglycerides. This results in building up of a large amount of fatty material in body organs, which include spleen, liver, and gut. The deficiency of LAL causes two autosomal recessive disorders, Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD). LAL deficiency is caused due to the genetic mutation in LIPA gene, which results in decrease or loss of Lysosomal Acid Lipase (LAL) enzyme.

FDA approval of innovative drugs is expected to fuel the growth of Lysosomal Acid Lipase (LAL) Deficiency Treatment Market

The approval of innovative drugs by Food and Drug Administration (FDA) is expected to augment the growth of market. In 2015 the U.S. Food and Drug Administration (FDA) approved Alexion Pharmaceutical’s Kanuma (sebelipase alfa), an innovative enzyme replacement therapy (ERT). Kanuma is first approved therapy that treats the underlying cause of the disease. It is the only therapy for the treatment of LAL deficiency. As it is the only available therapy in market, kanuma is expected to have a significant market growth in the near future. According to the National Institute for Health and Care Excellence (NICE) 2016, around 50% of patients suffering from lysosomal acid lipase (LAL) deficiency have chances of liver complications, which includes cirrhosis or fibrosis and liver transplant. The increasing incidences of liver complications due to LAL deficiency is expected to increase demand for liver transplant, in turn fueling the growth of LAL deficiency treatment market over the forecast period. High cost and lack of awareness about diagnosis and treatment among people is the major restraint for the growth of Lysosomal Acid Lipase (LAL) Deficiency treatment market.

Lysosomal Acid Lipase (LAL) Deficiency Treatment Market Taxonomy:

The global Lysosomal Acid Lipase (LAL) deficiency treatment market is segmented on the basis of disease type, treatment type, and end user.

On the basis of disease type, the Lysosomal Acid Lipase (LAL) deficiency treatment market is segmented into:

  • Wolman Disease
  • Cholesteryl Ester Storage Disease (CESD)

On the basis of treatment type, the Lysosomal Acid Lipase (LAL) deficiency treatment market is segmented into:

  • Liver Transplant
  • Hematopoietic Stem Cell Transplant

On the basis of end users, the Lysosomal Acid Lipase (LAL) deficiency treatment market is segmented into:

  • Hospital
  • Clinics

North America is expected to dominate market due to rapid novel innovations

Global market for the Lysosomal Acid Lipase (LAL) deficiency treatment market by Coherent Market Insights is segmented into North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America is expected to dominate the lysosomal acid lipase (LAL) deficiency treatment market attributed to rapid novel innovations, drug development, and research. In 2015, FDA approved genetically modified organisms (GMO) chickens, for the production of a recombinant human lysosomal acid lipase (LAL) protein in their eggs for the treatment of lysosomal acid lipase (LAL) deficiency among Americans. Europe is expected to be the second largest region for LAL deficiency treatment market. The Europe commission in 2015 granted marketing permission of Kanuma, for the treatment of LAL deficiency for patients of all ages, which is expected to fuel the growth of kanuma in region.

Rapid approval of therapy by FDA in various region, be an opportunities for companies

Ltd. In 2015, FDA approved Kanuma, an innovative enzyme replacement therapy (ERT) in the U.S., Europe Union, and Japan, which is providing an opportunity to manufacturers for exploring their business in the untapped market. Major players operating in global Lysosomal Acid Lipase (LAL) Deficiency treatment market include Alexion Pharmaceutical Inc., Merck & Co., Inc., Pfizer, Inc., and AstraZeneca plc. and Teva Pharmaceutical Industries.


Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.

Data Triangulation Methodology | Coherent Market Insights

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.

Market Analysis | Coherent Market Insights

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users