PUBLISHER: Grand View Research | PRODUCT CODE: 1630991
PUBLISHER: Grand View Research | PRODUCT CODE: 1630991
The global lysosomal acid lipase deficiency treatment market size is expected to reach USD 433.2 million by 2030, registering a CAGR of 7.2% from 2025 to 2030, according to a new report by Grand View Research, Inc. High unmet needs, rising prevalence, and increasing uptake of enzyme replacement therapy are some of the primary factors driving the 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 a 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 the 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 the goal to overcome resistance and minimize toxicity.