Lysosomal Acid Lipase Deficiency Treatment Market - By Indication, By Treatment, By Geography - Global Opportunity Analysis & Industry Forecast, 2024 - 2030

Lysosomal Acid Lipase Deficiency Treatment Market Overview

Lysosomal Acid Lipase Deficiency Treatment Market Size is forecast to reach $ 445 Million by 2030, at a CAGR of 7.00% during forecast period 2024-2030. Lysosomal acid lipase deficiency is a disease/condition that affects the cholesterol metabolism of a young or an adult. Moreover, it is a genetic disorder that is primarily caused due to mutation in the LIPA gene. As a result of having this disease, one will have substantially high amounts of triglycerides or would develop cholesteryl esters in different parts of their body. Moreover, with the rising fats and other cholesterol, developing cardiovascular diseases is incredibly high. However, the disease remains undiagnosed for a long time, as there are no apparent symptoms of it as such. Additionally, the prevalence of the following disease is between 1:40,000 to 1:300,000.

The increase in the diagnostic application and technology, along with the knowledge for early detection of the associated diseases, are some of the factors driving the Lysosomal Acid Lipase Deficiency Treatment Market forward in the projected period of 2021-2026.

Report Coverage

The report: “Lysosomal Acid Lipase Deficiency Treatment Market Forecast (2024-2030)”, by Industry ARC covers an in-depth analysis of the following segments of the Lysosomal Acid Lipase Deficiency Treatment Market.

By Indication- Wolman Disease and Cholesteryl Ester Storage Disease.

By Treatment- Enzyme Replacement Therapy, Lipid Modifying Agents, Stem Cell Therapy, Surgery, and Supportive Care.

By Geography- North America (U.S., Canada, Mexico), Europe (Germany, United Kingdom (U.K.), France, Italy, Spain, Russia, and Rest of Europe), Asia Pacific (China, Japan India, South Korea, Australia, and New Zealand, and Rest of Asia Pacific), South America (Brazil, Argentina, and Rest of South America), and Rest of the World (the Middle East, and Africa).

Key Takeaways

• Geographically, North America’s Lysosomal Acid Lipase Deficiency Treatment Market held a dominant share in the year 2020. It is owing to the prevalence of the following auto-immune disease in the area alongside, and the FDA is readily trying to roll out therapy to cure the disease and reduce mortality. However, Asia-Pacific shows lucrative growth opportunities for the marketers owing to a steady increase in the R&D facilities in Japan and Korea to find alternative and efficient diagnostic systems.
• The rapid approval and readiness by various country’s health bodies have acted as a market driver. However, the prevalence of the disease is on the lower side, and hence the market’s potential to expand holistically is less.
• A detailed analysis of the strengths, weaknesses, opportunities, and threats will be provided in the Lysosomal Acid Lipase Deficiency Treatment Market.

Lysosomal Acid Lipase Deficiency Treatment Market- Geography as (%) for 2020.

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Lysosomal Acid Lipase Deficiency Treatment Market Segmentation Analysis- By Indication

The Lysosomal Acid Lipase Deficiency Treatment Market based on indication can be further segmented into Wolman Disease and Cholesteryl Ester Storage Disease. Wolman disease held a dominant market share in the year 2020. It is owing to its high prevalence at birth and with no apparent symptoms as well. Additionally, the indication is only visible post diarrhea, vomiting, and acute form of jaundice. Moreover, the fatality due to the following sign is relatively higher close to 1 in 3 children fails to make it through. Additionally, the affected children showed severe malnutrition, adrenal calcifications, and moreover deaths.

However, CESD or Cholesteryl Ester Storage Disease is estimated to be the fastest-growing, with a CAGR of 7.00% over the forecast period of 2024-2030. The following is the condition that is felt when the genetic disorder only shows up in childhood and not during the phase of an infant. Moreover, the situation is less severe than the former, as the chance of fatality reduces to 1 in 10, and that too even after developing any cardiovascular diseases because of high triglycerides. Moreover, the condition can be diagnosed till the age of 40.

Lysosomal Acid Lipase Deficiency Treatment Market Segmentation analysis- By Treatment

The Lysosomal Acid Lipase Deficiency Treatment Market based on treatment can be further segmented into Enzyme Replacement Therapy, Lipid Modifying Agents, Stem Cell Therapy, Surgery, and Supportive Care. The lipid-modifying agents held a dominant market share in the year 2020. It is owing to their low cost and usage across the age group to assist in cholesterol metabolism. For example, Ezetimibe and statin as the modifying agents have been used as a combination that has helped achieve favorable results at a much faster rate than monotherapy. As per a study conducted amongst 1500 patients, the LDL-C < 100 and < 70 mg/Dl when the patients were given Ezetimibe and statin, however a higher titration of mono therapeutic drug known as rosuvastatin provided with the same result in a higher duration of time.

However, enzyme replacement therapy is estimated to be the fastest-growing, with a CAGR of 7.00% over the forecast period of 2024-2030. It is owing to optimal results post such procedure than the other methods. In the following procedure, the replacement enzymes are given to the affected patient via IV infusions. Moreover, by receiving these enzymes, the body can provide a function which it was not able to do before.

Lysosomal Acid Lipase Deficiency Treatment Market Segmentation analysis- By Geography

The Lysosomal Acid Lipase Deficiency Treatment Market market based on Geography can be further segmented into North America, Europe, Asia-Pacific, South America, and the Rest of the World. North America’s region held a dominant market share of 36% as compared to the other regions. It is owing to the ready adoption of the FDA to regulate more and more drugs to help in cholesterol management and overall prevention of developing severity due to cardiovascular diseases. FDA has approved six drugs in the past 1.5 years related to cholesterol management and cultured enzyme medicines.

Moreover, the Asia-Pacific region is set to offer lucrative growth opportunities to marketers. It is owing to the recent technological diagnostic advancement in the countries such as Korea and Japan, which have made diagnostic procedures more accessible, faster, and reliable. Furthermore, the tests error ratio has been tried to be bought down below 3% on average.

Lysosomal Acid Lipase Deficiency Treatment Market Drivers

The technological advancement in diagnostic procedures is driving the market forward.

The most prominent methodology to detect the LAL deficiency was done by genetic mutation testing along with identifying the mutations of the LIPA gene. Moreover, the enzymes were collected and measured in the cultured fibroblasts, peripheral leukocytes, or even liver tissue. However, the test results varied between untestable and 16% of the normal as per the assays used. Moreover, the assays used could not help in noticing the disease severity, and even the lab results across the state couldn’t be compared. But, for now, various new technology has been launched to determine the LAL activity. The technology uses “dried blood spots.” Additionally, Lalistat is used as it is a highly specific inhibitor of the LAL. Therefore, a cross-comparison can be drawn, as the total lipase activity can be differentiated with the lipase activity with Lalistat 2.

Additionally, the technology offers benefits such as small sample size, specimen transport, as stable temperature. Furthermore, LIPA gene analysis is also helpful in the diagnosis of LAL-D. To date, over 40 loss-of-function LIPA mutations have been identified.

The management techniques have advanced and are a driving factor for the market.

HMG-CoA was predominantly used in various clinical research, and in one of the trials, the lipid parameters improved, and additionally, reduction in cholesterol synthesis and decreased secretion of apo B-containing lipoproteins were observed. But the studies didn’t show many improvements when the trial was conducted on others. For 12 people given the HMG-CoA, no improvement was seen. Only later, the FDA agreed to launch the first-ever treatment known as Sebi lipase alfa, a recombinant human enzyme replacement. The medication helped in reducing the LDL-C by 28% in the control group as with 6% of the placebo group. Moreover, the cases of anaphylaxis have been bought down by up to 2% in all the IV treatments provided. Thus, the following medication has reduced the fatality by a considerable amount, and moreover, the management within the infants, adults, and children has improved.

Lysosomal Acid Lipase Deficiency Treatment Market Challenges

The prevalence of the condition is rare, and the treatment costs are high; hence, the market reach would be limited. 

Since the following condition is extremely rare and is only found in between 1:40,000 to 1:300,000, the market participants cannot run deep and vast. Moreover, the people have less idea about such treatments, and the cost for the same can run extremely high and dent heavily. For example, a 2Mg/ML Kanuma intravenous solution can cost up to $11,000 for 10 milliliters, and the price per unit is close to $1,067.

Lysosomal Acid Lipase Deficiency Treatment Market Competitive Landscape

Product launches, mergers and acquisitions, joint ventures, and geographical expansions are key strategies adopted by players in the Lysosomal Acid Lipase Deficiency Treatment Market. The top 10 Lysosomal Acid Lipase Deficiency Treatment Market companies include:

1. Pfizer Inc.
2. Merck.
3. Alexion Pharma
4. Novartis
5. Vytorin
6. Mevacor
7. Crestor
8. Zocor
9. Pravachol
10. Livalo 

Recent Developments

• In April 2021, the FDA approved the drug Praluent, which is given in the form of an injection. Moreover, it helps lower genetically high forms of cholesterol or people affected with HoFH and LAL deficiency.
• In June 2020, FDA approved two new non-statin drugs known as Nexletol and Nexlizet. Both the medication helps in treating high cholesterol caused due to genetic moderations and can be moreover used in generic cases well.

Key Market Players:

The Top 5 companies in the Lysosomal Acid Lipase Deficiency Treatment Market are:

1. Pfizer Inc.
2. Merck & Co.
3. Regeneron Pharmaceuticals, Inc.
4. AstraZeneca
5. Myriad Genetics Inc.

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