Lysosomal disease is caused due to deficiency or absence of lysosomal acid lipase enzyme. The deficiency causes structural abnormalities of lysosome, result in storage of several toxic substances in the body cells. There are about 50 different types of these illnesses, which can harm heart, brain, skin, skeleton, and central nervous system among other physical organs. More lysosomal diseases are continuously being identified. Many lysosomal diseases currently lack an approved drug, despite the fact that clinical trials for prospective therapies for some of these disorders are now in progress.
A set of inherited metabolic diseases known as lysosomal storage disorders are brought on by enzyme deficits in different body cells. Key driver for growth of the global lysosomal disease treatment market includes increasing diagnosis rate, owing to increase in awareness and financial incentives for orphan drug development, which treats lysosomal diseases. In addition, rise in prevalence of lysosomal diseases, affordable product cost, increase in pharmaceutical companies for development of novel medications to treat lysosomal diseases are also responsible for growth of the market. Lack of treatment for lysosomal diseases and high cost of treatments are the factors that restrain growth of the market.
The lysosomal disease treatment market is segmented on the basis of disease type, type of therapy, end user, and region. By disease type, the market is divided into Gaucher’s diseases, Fabry diseases, Pompe’s syndrome, Mucopolysaccharidosis, and others. The others segment is further classified into aspartylglucosaminuria, batten disease, cystinosis, and glycogen storage disease II. By type of therapy, the market is fragmented into Enzyme replacement therapy, Stem cell therapy, Substrate reduction therapy, and others. By end user, it is segmented into Hospitals, Clinics, and others. Region wise, the market is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Major key players that operate in the global lysosomal disease treatment market include Amicus Therapeutics, Inc, Alexion Pharmaceuticals, Inc, BioMari, Chiese Farmaceutici SpA, Eli Lilly Company, Horizon Therapeutics Plc, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Leadient Biosciences, Inc Novartis AG, Orphazyme A/S, Pfizer Inc, Recordati SpA, Sanofi (Genzyme Corporation), Sigilon Therapeutics, Inc, and Takeda Pharmaceutical Company Limited (Shire Plc) Valerion Therapeutics, Llc, and Viatris Inc (Mylan NV).
KEY BENEFITS FOR STAKEHOLDERS
- This report provides a quantitative analysis of the market segments, current trends, estimations, and dynamics of the lysosomal disease treatment market analysis from 2021 to 2031 to identify the prevailing lysosomal disease treatment market opportunities.
- The market research is offered along with information related to key drivers, restraints, and opportunities.
- Porter's five forces analysis highlights the potency of buyers and suppliers to enable stakeholders make profit-oriented business decisions and strengthen their supplier-buyer network.
- In-depth analysis of the lysosomal disease treatment market segmentation assists to determine the prevailing market opportunities.
- Major countries in each region are mapped according to their revenue contribution to the global market.
- Market player positioning facilitates benchmarking and provides a clear understanding of the present position of the market players.
- The report includes the analysis of the regional as well as global lysosomal disease treatment market trends, key players, market segments, application areas, and market growth strategies.
Key Market Segments
By Disease Type
- Gaucher's Diseases
- Fabry Diseases
- Pompe’s Syndrome
- Mucopolysaccharidosis
- Others
By Type of Therapy
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Substrate Reduction Therapy
- Others
By End User
- Hospitals
- Clinics
- Others
By Region
- North America
- U.S.
- Canada
- Mexico
- Europe
- Germany
- France
- U.K.
- Italy
- Spain
- Rest of Europe
- Asia-Pacific
- Japan
- China
- Australia
- India
- South Korea
- Rest of Asia-Pacific
- LAMEA
- Brazil
- Saudi Arabia
- South Africa
- Rest of LAMEA
Key Market Players
- Amicus Therapeutics, Inc.
- Alexion Pharmaceuticals, Inc
- BioMarin
- Johnson & Johnson
- Novartis AG
- Pfizer Inc.
- Sanofi
- Takeda Pharmaceutical Company Limited
- Eli Lilly and Company
- Sigilon Therapeutics, Inc
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Table of Contents
Executive Summary
According to the report, titled, “Lysosomal Disease Treatment Market," the lysosomal disease treatment market was valued at $7.6 billion in 2021, and is estimated to reach $14.1 billion by 2031, growing at a CAGR of 6.4% from 2022 to 2031.Lysosomal disease (LD) is a hereditary metabolic disorder brought on by inadequate lysosomal enzyme synthesis. Human body contains over 50 different forms of lysosomal storage disorders. In addition, there are few choices for treating the illness. The lysosomal disease treatment market share is expected to expand as more research and development (R&D) activities are conducted in an effort to create an effective treatment. Expanding government programs and investments for development of innovative medications is expected to boost the market growth during the forecast period. Moreover, rise in prevalence of lysosomal disease treatment is expected to boost the lysosomal disease treatment market growth. Introduction of orphan medicines is also anticipated to fuel the market expansion.
Growth in awareness regarding preventative measures for lysosomal diseases and lysosomal disease treatment market trends are anticipated to propel the growth of lysosomal disease treatment market size. The lysosomal disease treatment market is also expected to grow, owing to expansion of pharmaceutical and healthcare industries. However, lack of effective treatments and their high-cost are the factors restrain the market growth. The demand for lysosomal disease treatment significantly decreased after the COVID-19 pandemic. The focus of medical professionals, governments, and general public has moved to containing spread of the virus as a result of quick expansion of the virus. It consequently decreased the need for lysosomal disease treatment. People also stopped going to clinics and hospitals unless they had an emergency. As a result, the COVID-19 pandemic caused severe disruptions in the market for treating lysosomal diseases. Lysosomal disease is group of inherent diseases affects the normal body fuctions of brain, skeletal and central nervous system. During the covid-19 period the need for lysosomal disease treatment decreased significantly. However, the diagnostic methods used for the lysosomal diseases are enzyme assay activity, optical fluorometry, mutation scanning, mass spectroscopy and DNA sequencing. In various hospitals screening of newborn for various test including lysosomal disease test is mandatory. According to the U.S. National Library of Medicine 1 in 40,000 to 60,000 males have Fabry's disease. Pompe’s syndrome is affecting about 1 in 40,000 people in the U.S. according to the National Organization for Rare Disorders. Moreover, Pompe’ syndrome affects about 1 in 40,000 people in the U.S., according to the National Organization for Rare Disorders
Coronavirus Severe acute respiration syndrome (SARS-CoV-2) is an infectious disease caused by the novel coronavirus (COVID-19), which originated in the Wuhan district in China in the late 2019, and since has spread to 212 countries. The virus was initially referred to as “novel coronavirus 2019” (2019-nCoV) by the WHO, However, on February 11, 2020, it was given the official name of SARS-CoV-2 by the International Committee on Taxonomy of Viruses. WHO declared COVID-19 as pandemic on March 11, 2020, and by September 1, 2020, over 28.1 million people have been infected globally with over 909,000 deaths. COVID-19 symptoms include fever, cough, and shortness of breath.
Nearly all industries have been impacted by the COVID-19. As coronavirus crises sweep the globe and force healthcare organizations to devote majority of their funds to fighting COVID-19, the outbreak of COVID-19 has resulted in a significant decline in demand for the lysosomal disease treatment across several sectors, particularly the health and pharmaceutical sector. The main cause of the interruptions experienced by patients getting treatment for lysosomal storage disorder in hospitals was the risk of infection. As a result, the COVID-19 outbreak is expected to slow the global market growth for treatments for lysosomal diseases, which will have a negative effect on the market's value in 2022 and beyond
By disease type, the Gaucher's disease segment dominated the market in 2021, and is expected to remain dominant during the forecast period, owing to increase in incidences of lysosomal diseases and rise in awareness related to early diagnosis of lysosomal diseases.
Depending on type of therapy, the enzyme replacement therapy segment dominated the market in 2021, and is expected to remain dominant during the forecast period, owing to increasing research and development (R&D) activities to develop an efficient treatment.
On the basis of end user, the hospitals segment dominated the market in 2021, and is expected to remain dominant during the forecast period, owing to rise in rare diseases and increase in success rate of treatment of lysosomal diseases.
Region wise, North America has the highest lysosomal disease treatment industry share in 2021. Furthermore, availability of products for treatment of lysosomal diseases and high disease prevalence are the major factors responsible for the market growth of this region.
Key Findings of the Study
- By disease type, the Gaucher's diseases segment dominated the market in 2021, and it is expected to remain dominant throughout the forecast period.
- On the basis of type of therapy, the enzyme replacement therapy segment dominated the market in 2021.
- By end user, the hospitals segment dominated the market in 2021.
- Region wise, North America dominated the market in 2021.
Companies Mentioned
- Amicus Therapeutics, Inc.
- Alexion Pharmaceuticals, Inc
- Biomarin
- Johnson & Johnson
- Novartis AG
- Pfizer Inc.
- Sanofi
- Takeda Pharmaceutical Company Limited
- Eli Lilly and Company
- Sigilon Therapeutics, Inc
Methodology
The analyst offers exhaustive research and analysis based on a wide variety of factual inputs, which largely include interviews with industry participants, reliable statistics, and regional intelligence. The in-house industry experts play an instrumental role in designing analytic tools and models, tailored to the requirements of a particular industry segment. The primary research efforts include reaching out participants through mail, tele-conversations, referrals, professional networks, and face-to-face interactions.
They are also in professional corporate relations with various companies that allow them greater flexibility for reaching out to industry participants and commentators for interviews and discussions.
They also refer to a broad array of industry sources for their secondary research, which typically include; however, not limited to:
- Company SEC filings, annual reports, company websites, broker & financial reports, and investor presentations for competitive scenario and shape of the industry
- Scientific and technical writings for product information and related preemptions
- Regional government and statistical databases for macro analysis
- Authentic news articles and other related releases for market evaluation
- Internal and external proprietary databases, key market indicators, and relevant press releases for market estimates and forecast
Furthermore, the accuracy of the data will be analyzed and validated by conducting additional primaries with various industry experts and KOLs. They also provide robust post-sales support to clients.
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Table Information
Report Attribute | Details |
---|---|
No. of Pages | 319 |
Published | July 2022 |
Forecast Period | 2021 - 2031 |
Estimated Market Value ( USD | $ 7572.97 million |
Forecasted Market Value ( USD | $ 14090.39 million |
Compound Annual Growth Rate | 6.4% |
Regions Covered | Global |
No. of Companies Mentioned | 10 |