The December 2015 U.S. FDA approval of a breakthrough drug in the LAL deficiency treatment market, has set the ball rolling for tremendous potential in the growth of this rare-disease market.
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Lysosomal Acid Lipase (LAL) Deficiency, also known as Cholesterol Ester Storage Disorder (CESD), Wolman’s Disease or LAL deficiency, is an inherited, ultra-rare genetic disease affecting children as well as adults. Lysosomal acid lipase (LAL) is responsible for the hydrolysis of cholesterol esters and triglycerides. Patients with LAL deficiency have no or little LAL enzyme activity which results in the accumulation of fats within various body cells that leads to high cholesterol and triglycerides, and a very low HDL-cholesterol level. There are two main types of LAL disorder: early onset Wolman Disease and late onset CESD. Wolman disease is very rare, with an incidence of less than one in 100,000 live births. CESD is a less severe, later-onset disorder which can occur in adults at any stage in life, having an incidence of about one in 25 per million births. While Wolman disease is a relatively occurrence, the infants affected with it usually die within the first year of birth after experiencing growth failure, liver fibrosis, and cirrhosis. In late onset LAL deficiency, the accumulation of cholesteryl esters and triglycerides in blood vessels, vital organs, and other tissues of adults, results in progressive and multiple-organ damage which includes fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, and cardiovascular disease. Since LAL disease has symptoms similar to many other diseases, for instance, heart disease, it is often misdiagnosed. The disease in its late onset form does not affect everyone equally and thus symptoms can vary from person to person, often manifesting as mild symptoms which consequently also makes the disease underdiagnosed worldwide. The biggest factor that shall drive the market is that currently, apart from a recently approved drug by U.S. FDA known as Kanuma (sebelipase alfa), there is no approved treatment available for the disease. Thus, tremendous business opportunity lies in the investment of finding therapies for treatment in this market. Lack of awareness regarding disease diagnosis and untimely treatment due to ignorance of symptoms may restrain the growth of the market.
The global market for lysosomal acid lipase (LAL) deficiency is segmented on basis of type of therapy, type of drug, disease indication and geography:
Segmentation by Type of Therapy (no. of surgeries)
- Liver Transplant
- Hematopoietic Stem Cell Transplant
Segmentation by Type of Drug
- Statins and other dyslipidemia drugs
- Sebelipase alfa (Kanuma)
Segmentation by Disease Indication
- Wolman Disease
- Cholesteryl ester storage disease (CESD)
LAL deficiency is a chronic progressive genetic metabolic disease associated with substantial morbidity as well as premature mortality. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population. The current management practice for the disease involves taking lipid lowering medications such as statins, to overcome symptoms of lipid accumulation. However, progression of the disease often necessitates the patient to undergo a liver transplant. The market for lysosomal acid lipase (LAL) deficiency is segmented based on number of surgeries: for liver transplant and for hematopoietic stem cell transplant. A liver transplant may give LAL patients respite from liver failure, but it does not tackle the underlying cause of the disease and may not stop disease progression in extra-hepatic organs, which include the heart and kidneys. However, it is still an option for patients who wish to increase chances of survival. Although hematopoietic stem cell transplant has been performed in a few infants with lysosomal acid lipase (LAL) deficiency, it has been associated with complications and the long term outcomes are unknown. Thus, the liver transplant segment shall witness more number of surgeries during the forecast period compared to the hematopoietic stem cell transplant segment.
Based on disease indication, sebalipase alfa segment is expected to contribute largest share in global lysosomal acid lipase (LAL) deficiency treatment market due to it being the only approved mainstay treatment for the disease. Statins although helpful in symptom management for lysosomal acid lipase (LAL) deficiency, are not effective in completely preventing the progression of the disease, resulting in their estimated lower share in the market compared to sebalipase alfa. Moreover, the value garnering segment would be sebalipase alfa due to its higher cost of treatment and also being the preferred treatment due to it being the only approved drug for this indication. On the basis of disease indication, the market has been segmented into wolman disease and cholesteryl ester storage disease (CESD). The market for CESD is anticipated to be higher due to comparatively higher need in this segment.
On the basis of regional presence, global lysosomal acid lipase (LAL) deficiency treatment market is segmented into five key regions viz. North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America and Europe are expected to lead the global lysosomal acid lipase (LAL) deficiency treatment market due to high prevalence of this rare disease.
Some of the major players in global lysosomal acid lipase (LAL) deficiency treatment market are Alexion Pharmaceutical Inc., AstraZeneca plc., Merck & Co., Inc., Pfizer, Inc. and Teva Pharmaceutical Industries Ltd.
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