Lysosomal Acid Lipase Deficiency Treatment Market
Lysosomal acid lipase (LAL) deficiency is a rare, chronic, progressive inherited disorder that affects the body’s ability to create an enzyme called lysosomal acid lipase (LAL), which is needed for breaking down of fats like cholesteryl ester and triglycerides. This causes buildup of fats in body organs including liver, gut and spleen in large amount. Wolman disease (WD) and Cholesteryl Ester Storage Disease (CESD) are the two recessive diseases that occur due to deficiency of lysosomal acid lipase. The genetic mutation in LIPA gene is responsible for deficiency of LAL.
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Frequent approval of new drugs is expected to propel growth of the global the lysosomal acid lipase (LAL) deficiency treatment market. For instance, in 2015, the U.S. FDA granted approval to Alexion Pharmaceutical’s Kanuma (sebelipase alfa). It is the first approved therapy for the treatment of LAL deficiency.
Rise in the cases of liver complications caused by LAL deficiency is likely to drive the demand of liver transplant, which in turn is expected to propel growth of the lysosomal acid lipase (LAL) deficiency treatment market during the forecast period. For instance, in 2016, the National institute for health and care excellence (NICE) reported that around 50% of people affected by LAL deficiency are at risk of developing liver complications including liver transplant and cirrhosis or fibrosis.
Lack of awareness among people regarding diagnosis and treatment as well as high cost of the treatment are the factors expected to hamper growth of the global the lysosomal acid lipase (LAL) deficiency treatment market.
The global lysosomal acid lipase (LAL) deficiency treatment market is regionally divided into North America, Europe, Latin America, Middle East, Asia Pacific and Africa.
Among regions, North America is expected to witness strong growth in the lysosomal acid lipase (LAL) deficiency treatment market, owing to increasing advancement of technology, development of drugs and research. For instance, in 2015, the U.S. FDA granted approval to genetically modified organisms (GMO) chickens, to produce a recombinant human lysosomal acid lipase (LAL) protein in their eggs for treating LAL deficiency in American population.
Europe is also expected to witness robust growth in the lysosomal acid lipase (LAL) deficiency treatment market, owing to approval of drugs for the treatment of LAL deficiency in patients of all age groups. For instance, in 2015, the European commission approved marketing permission of Kanuma for treating LAL deficiency in the region.
The global Lysosomal Acid Lipase (LAL) deficiency treatment market is divided into disease type, treatment type, and end user.
By disease type:
- Wolman Disease
- Cholesteryl Ester Storage Disease (CESD)
By treatment type:
- Liver Transplant
- Hematopoietic Stem Cell Transplant
By end users:
Key players functioning in the global Lysosomal Acid Lipase (LAL) Deficiency treatment market are Merck & Co., Inc., and AstraZeneca plc. Teva Pharmaceutical Industries, Alexion Pharmaceutical Inc., and Pfizer, Inc.,
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