Lysosomal Acid Lipase Deficiency – Pipeline Review, 2019

Lysosomal acid lipase deficiency refers to an inherited medical condition, which is characterized by the problems in the breakdown and usage of cholesterol and fats in the body or lipid metabolism. In the individuals affected with the disorder, there is an accumulation of harmful amounts of fats or lipids in the cells and throughout the body, subsequently resulting in liver disease. There are two forms of the disease: the severe and the rarest form of the disorder begins in infancy and the less severe form can begin from childhood to late adulthood. In the severe form of the disease, the accumulation of lipids begins in the first few weeks of life and these lead to several health problems. Infants with this form of the disease, generally do not survive past the first year of life.

Some of the common signs and symptoms of lysosomal acid lipase deficiency include abdominal pain and bloating, jaundice, hepatic fibrosis, adrenal calcification, and high liver enzymes. The prevalence of the disorder has been estimated to range from 1 in 40,000 to 1 in 300,000, depending on the ethnicity and geographical location of the individual. In terms of treatment and management of the disorder, there was no cure approved until 2015. In 2015, an enzyme replacement therapy called sebelipase alfa was approved in the U.S. and Europe. In Japan, sebelipase alfa was approved in 2016.

Pharmaceutical companies along with various research institutes have been focusing on studying and developing new treatment options for lysosomal acid lipase deficiency. For instance; human placental-derived stem cell, which is being studied by New York Medical College, is currently in phase-1 clinical trials for the human placental-derived stem cell transplantation (HPDSC).

At present around 70% of the pipeline candidates for lysosomal acid lipase deficiency are in the phase-2 stage. More than half of the studies are sponsored by industry.

Report Description

The report on ‘Lysosomal Acid Lipase Deficiency – Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Lysosomal Acid Lipase Deficiency. The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Lysosomal Acid Lipase Deficiency.

The report on ‘Lysosomal Acid Lipase Deficiency – Pipeline Review, 2019’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.

Report Scope

• Thorough assessment of the pipeline products by areas such as development stage; route of administration; drug class; indication; sponsor; molecule type and drug target


• Comprehensive profiles of the pipeline products with details such as company overview; product description; R&D status; development activities; mechanism of action; molecule type; development stage; indications; funding and route of administration


• Overview of dormant and discontinued pipeline products


• Key insights in relation to the epidemiology of conditions being treated by the pipeline products and overview of the addressable or current market for the pipeline products


• Overview of the latest developments; news articles, press releases and relevant conferences

Report Methodology

• All pipeline reports are built through the analysis of data primarily collected through credible desk research sources. Secondary research is supplemented by interviews conducted with key opinion leaders.


• Desk research sources includes global and regional clinical trials databases; annual reports, websites, press releases & investor presentations of companies; white papers; news articles; reports published by industry associations; articles/reports published on databases such as NCBI, ResearchGate; internal databases

Reasons to Buy this Report

• Develop effective growth strategies based on comprehensive overview of the R&D activity and pipeline products for Lysosomal Acid Lipase Deficiency


• Identify emerging players or competition in the market based on pipeline products and develop strategies to counter the emergence of these players


• Identify the focus of leading players in relation to R&D for Lysosomal Acid Lipase Deficiency


• Identify potential companies from a partnership or acquisition point of view based on current synergy in R&D activities or strategies to diversify R&D focus to drive growth in business


• Analyse the reasons behind dormant and discontinued products to make changes in the R&D focus if necessary