Lysosomal Acid Lipase Deficiency Treatment Market Size, Share & Trends Analysis Report By Treatment, By Indication (Wolman Disease, Cholesteryl Ester Storage Disease), And Segment Forecasts, 2018 - 2025

Industry Insights

The global lysosomal acid lipase (LAL) deficiency treatment market size was valued at USD 380 million in 2017. It is expected to exhibit a CAGR of 10.8% during the forecast period. Increasing uptake of enzyme replacement therapy, continued use of supportive care therapies, and high unmet needs are some of the key factors driving the market.

Lysosomal acid lipase deficiency (LAL-D) is an autosomal recessive genetic disorder affecting cholesterol metabolism in infants and adults. The disease is caused mainly due to a mutation in the lysosomal acid lipase (LIPA) gene, resulting in the accumulation of either triglyceride and/or cholesteryl esters in different tissues of the body.

LAL-D is an ultra-rare disease affecting less than 19,000 patients worldwide. Wolman disease affects two infants per million live births, most of who often die within a year of birth. Cholesterol Ester Storage Disease (CESD) is more prevalent and often affects children and adolescents. The disease is gender-neutral and patients present symptoms including hypercholesterolemia, hypertriglyceridemia, premature atherosclerosis, liver failure, and abnormal lipid deposition in organs such as the liver and spleen.

The symptoms of LAL deficiency, especially late-onset CESD often overlap with other liver-associated diseases such as nonalcoholic steatohepatitis, viral hepatitis, and autoimmune liver disease, resulting in misdiagnosis by physicians. Approval of first-ever ERT therapy for both WD and CESD has opened up a wave of opportunities for manufacturers to develop tailored therapies for this debilitating disease.

Indication Insights

Based on indication, the market has been bifurcated into Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD). Wolman disease is a more severe form and has an early onset. It occurs due to the nonexistence of LAL enzyme activity. Infants show no clinical signs at birth, but after a few months suffer from diarrhea, vomiting, jaundice, and develop enlarged livers and spleens. Furthermore, improper absorption of nutrients in the small intestine often results in extreme weight loss in patients.

CESD usually occurs among children and adolescents and has a higher prevalence compared to WD. The diagnosis is poor due to variations in the phenotype of patients and therefore, many cases remain unreported or undiagnosed. The CESD segment is estimated to expand at a higher CAGR during the forecast period, owing to the high disease prevalence and growing uptake of enzyme replacement therapy (ERT).

Treatment Insights

Current management of LAL deficiency is majorly focused on supportive care and nutritional support. Supportive care for patients includes management of plasma lipid levels, apolipoprotein B production, high cholesterol and triglycerides through lipid-lowering agents (statins), and a low-fat diet. Furthermore, the progressive nature of the disease often requires patients to undergo a hematopoietic stem cell transplant or liver transplant.

Based on treatment, the global LAL deficiency treatment market is segmented into enzyme replacement therapy, statins, surgery, stem cell transplant, and supportive care. Among these, the ERT segment is anticipated to post the highest CAGR during the forecast period, owing to high efficacy and long-term disease control after treatment. However, major challenges for the LAL-D treatment market remain low awareness and high treatment cost, which limits penetration and creates a financial burden for patients based in underdeveloped and developing markets.

Country Insights

The U.S. dominated the market in 2017, followed by Germany. The U.S. is poised to maintain its position in the global arena until 2023. The market is driven by the increasing uptake of Kanuma, high disease prevalence, and the presence of a large patient pool in the U.S.

Japan is projected to experience noteworthy growth during the forecast period, due to the approval of Kanuma and improvement in disease diagnosis.  The seven major markets together are likely to witness a higher growth rate than the rest of the world. There is a critical need for the development of a curative treatment approach that offers improved efficacy and the development of new early diagnostic markers to speed up disease diagnosis, worldwide.

LAL-D Treatment Market Share Insights

Currently, Alexion Pharma’s Kanuma is the only product approved for LAL-D treatment. Kanuma (sebelipase alfa) is a subcutaneously delivered enzyme replacement treatment(ERT) approved in the EU (September 2015), U.S. (December 2015), and Japan (March 2016) for LAL-D patients.

In addition, the CESD cohort is prescribed statins and other nutritional supplements. Pfizer (Lipitor) and Merck (Zetia) are key players in the statins segment of the CSED market. However, these companies are poised to lose market share on account of significant competition from generics and approval of ERT for both early and late-onset types of this disease.  Unmet needs within the LAL-D space present a strong opportunity to develop novel first-in-class therapies.

Report Scope

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of industry trends in each of the sub-segments from 2017 to 2025. For this study, Grand View Research has segmented the global LAL-D treatment market report based on indication, treatment, and country:

• Indication Outlook (Revenue, USD Million, 2017 - 2025)

  • Wolman Disease (WD)

  • Cholesteryl Ester Storage Disease (CESD)

• Treatment Class Outlook (Revenue, USD Million, 2017 - 2025)

  • Enzyme Replacement Therapy

  • Lipid-Modifying Agents (Statins)

  • Surgery (Liver Transplant)

  • Stem Cell Transplantation

  • Supportive Care

• Country Outlook (Revenue, USD Million, 2017 - 2025)

  • The U.S.

  • The U.K.

  • France

  • Germany

  • Italy

  • Spain

  • Japan