3-June-2019: 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.
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.
In
depth research report on Lysosomal Acid Lipase (LAL) Deficiency Treatment
Market
Further
key findings from the report suggest:
· 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.
Grand View Research has
segmented the global lysosomal acid lipase deficiency treatment marketbased 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
Browse
more research reports of this category:
About
Grand View Research
Grand View Research, Inc. is a
U.S. based market research and consulting company, registered in the State of
California and headquartered in San Francisco. The company provides syndicated
research reports, customized research reports, and consulting services. To help
clients make informed business decisions, the company offers market
intelligence studies ensuring relevant and fact-based research across a range
of industries including technology, chemicals, materials, healthcare and
energy.
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