Lysosomal
acid lipase (LAL) deficiency is a rare inherited condition in which
the body is not able to produce enough lysosomal acid lipase (LAL)
enzyme, which is an important enzyme for breakdown of fatty materials
namely cholesteryl esters and triglycerides. This results in building
up of a large amount of fatty material in body organs, which include
spleen, liver, and gut. The deficiency of LAL causes two autosomal
recessive disorders, Wolman disease (WD) and Cholesteryl Ester
Storage Disease (CESD). LAL deficiency is caused due to the genetic
mutation in LIPA gene, which results in decrease or loss of Lysosomal
Acid Lipase (LAL) enzyme.
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FDA
approval of innovative drugs is expected to fuel the growth of
Lysosomal Acid Lipase (LAL) Deficiency Treatment Market
The
approval of innovative drugs by Food and Drug Administration (FDA) is
expected to augment the growth of market. In 2015 the U.S. Food and
Drug Administration (FDA) approved Alexion Pharmaceutical’s Kanuma
(sebelipase alfa), an innovative enzyme replacement therapy (ERT).
Kanuma is first approved therapy that treats the underlying cause of
the disease. It is the only therapy for the treatment of LAL
deficiency. As it is the only available therapy in market, kanuma is
expected to have a significant market growth in the near future.
According to the National Institute for Health and Care Excellence
(NICE) 2016, around 50% of patients suffering from lysosomal acid
lipase (LAL) deficiency have chances of liver complications, which
includes cirrhosis or fibrosis and liver transplant. The increasing
incidences of liver complications due to LAL deficiency is expected
to increase demand for liver transplant, in turn fueling the growth
of LAL deficiency treatment market over the forecast period. High
cost and lack of awareness about diagnosis and treatment among people
is the major restraint for the growth of Lysosomal Acid Lipase (LAL)
Deficiency treatment market.
North
America is expected to dominate market due to rapid novel innovations
Global
market for the Lysosomal Acid Lipase (LAL) deficiency treatment
market by Coherent Market Insights is segmented into North America,
Latin America, Europe, Asia Pacific, Middle East, and Africa. North
America is expected to dominate the lysosomal acid lipase (LAL)
deficiency treatment market attributed to rapid novel innovations,
drug development, and research. In 2015, FDA approved genetically
modified organisms (GMO) chickens, for the production of a
recombinant human lysosomal acid lipase (LAL) protein in their eggs
for the treatment of lysosomal acid lipase (LAL) deficiency among
Americans. Europe is expected to be the second largest region for LAL
deficiency treatment market. The Europe commission in 2015 granted
marketing permission of Kanuma, for the treatment of LAL deficiency
for patients of all ages, which is expected to fuel the growth of
kanuma in region.
Rapid
approval of therapy by FDA in various region, be an opportunities for
companies
Ltd.
In 2015, FDA approved Kanuma, an innovative enzyme replacement
therapy (ERT) in the U.S., Europe Union, and Japan, which is
providing an opportunity to manufacturers for exploring their
business in the untapped market. Major players operating in global
Lysosomal Acid Lipase (LAL) Deficiency treatment market include
Alexion Pharmaceutical Inc., Merck & Co., Inc., Pfizer, Inc., and
AstraZeneca plc. and Teva Pharmaceutical Industries.
Lysosomal
Acid Lipase (LAL) Deficiency Treatment Market Taxonomy:
The
global Lysosomal Acid Lipase (LAL) deficiency treatment market is
segmented on the basis of disease type, treatment type, and end user.
On
The Basis Of Disease Type:
-
Wolman Disease
-
Cholesteryl Ester Storage Disease (CESD)
On
The Basis Of Treatment Type:
-
Liver Transplant
-
Hematopoietic Stem Cell Transplant
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