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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. 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.

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Lysosomal Acid Lipase Deficiency Treatment Market

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