SAN FRANCISCO, May 1, 2019 /PRNewswire/ -- The global lysosomal acid lipase deficiency (LAL-D) treatment market size is likely to reach USD 954 million by 2025, as per a new report by Grand View Research, Inc., exhibiting a 10.8% CAGR during the forecast period. High unmet needs, rising prevalence, and increasing uptake of enzyme replacement therapy are some of the primary factors driving the market.
Key suggestions from the report:
- CESD manifests later in life and is more prevalent among Caucasians and Hispanic populations, affecting approximately 1 in 300,000 individuals
- Germany is at the forefront of the Europe market and is driven by high disease prevalence and availability of drugs in the ERT class
- Approval of Kanuma in ERT class highlight a fundamental shift in treatment approach to LAL-D deficiency
- The ERT segment is estimated to be the fastest growing during the forecast period, owing to Kanuma's first-mover advantage as the only long-term treatment in untapped LAL-D patient population pool
- Statins are anticipated to lose market share due to rising competition from generics within the class and increasing uptake of Alexion's Kanuma
- Rising eligible patient population and limited therapeutic intervention for LAL deficiency offer strong commercial opportunity to players to invest in developing novel treatments in this market¬
- Lack of awareness in disease diagnosis and delayed access to medications due to poor prognosis may restrain the growth of the market.
Read 65 page research report with TOC on "Lysosomal Acid Lipase (LAL) Deficiency Treatment Market Size, Share & Trends Analysis Report By Treatment, By Indication (Wolman Disease, Cholesteryl Ester Storage Disease), And Segment Forecasts, 2018 - 2025" at: https://www.grandviewresearch.com/industry-analysis/lysosomal-acid-lipase-deficiency-lal-d-treatment-market
LAL deficiency (LAL-D) is a rare type of lysosomal storage disorder in which patients are unable to breakdown cholesteryl esters and triglycerides due to a mutation in the LAL gene. As a result, there is an accumulation of lipids in the liver, spleen, and walls of blood vessels. LAL deficiency is a progressive disease with multi-systemic clinical manifestations, significant medical complications, and early mortality due to liver failure, especially among infants.
Until 2015, the global LAL deficiency treatment market witnessed modest growth due to lack of approved treatments for the disease. Medical care was limited to supportive care, with surgery and lipid-modifying drugs such as statins remained the mainstay for treatment. In 2015, approval of Alexion Pharma's Kanuma (sebelipase alfa) marked entry of enzyme replacement therapy (ERT) in the LAL-D space. Kanuma is a hydrolytic lysosomal cholesterol ester and triacylglycerol specific enzyme, administered intravenously in patients with LAL deficiency. Currently, Kanuma is the only drug approved for both Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD) in the key markets.
Based on treatment, supportive care was the leading revenue contributor in the market in 2017. However, the ERT segment is expected to register the fastest growth during the forecast period, supported by continued uptake of Kanuma. Disease prognosis remains poor, as a majority of patients are either undiagnosed or misdiagnosed. Thus, there remains a strong need for extensive research to understand disease biology and develop effective therapeutic options with goal to overcome resistance and minimize toxicity.
Grand View Research has segmented the global lysosomal acid lipase deficiency treatment market based on indication, treatment, and country:
- Lysosomal Acid Lipase Deficiency Indication Outlook (Revenue, USD Million, 2017 - 2025)
- Wolman Disease (WD)
- Cholesteryl Ester Storage Disease (CESD)
- Lysosomal Acid Lipase Deficiency Treatment Outlook (Revenue, USD Million, 2017 - 2025)
- Enzyme Replacement Therapy (ERT)
- Lipid-Modifying Agents (Statins)
- Surgery
- Stem Cell Transplantation
- Supportive Care
- Lysosomal Acid Lipase Deficiency Country Outlook (Revenue, USD Million, 2017 - 2025)
- U.S.
- U.K.
- France
- Germany
- Italy
- Spain
- Japan
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About Grand View Research
Grand View Research, U.S.-based market research and consulting company, provides syndicated as well as customized research reports and consulting services. Registered in California and headquartered in San Francisco, the company comprises over 425 analysts and consultants, adding more than 1200 market research reports to its vast database each year. These reports offer in-depth analysis on 46 industries across 25 major countries worldwide. With the help of an interactive market intelligence platform, Grand View Research helps Fortune 500 companies and renowned academic institutes understand the global and regional business environment and gauge the opportunities that lie ahead.
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