Global Lysosomal Disease Treatments Market, Type (Fabry Disease, Tay - Sachs disease, and Others), Treatment (Enzyme Replacement Therapy, Stem Cell Transplantation, and Others), Route of Administration (Oral, Parenteral, and Others), End-Users (Hospitals, Specialty Clinics, Homecare, and Others), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others) – Industry Trends and Forecast to 2030.
Lysosomal Storage Disorder Drugs Market Analysis and Size
Lysosomal storage disorders are inherited metabolic disorders marked by an abnormal accumulation of numerous harmful chemicals in the body's cells due to enzyme shortages. There are roughly 50 of these ailments, and they can affect the skeleton, brain, skin, heart, and central nervous system, among other bodily components. There are still more lysosomal storage disorders being discovered. While clinical trials for potential treatments for some of these disorders are underway, many lysosomal storage diseases lack an approved medication.
Data Bridge Market Research analyses that the lysosomal storage disorder drugs, which was USD 9738.63 million in 2022, would rocket up to USD 21646.92 million by 2030 and is expected to undergo a CAGR of 10.50% during the forecast period. “enzyme replacement therapy” dominates the treatment segment of the lysosomal storage disorder drugs owing to rising prevalence of lysosomal disease in addition to the insights on market scenarios such as market value, growth rate, segmentation, geographical coverage, and major players, the market reports curated by the Data Bridge Market Research also include depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.
Lysosomal Storage Disorder Drugs Market Scope and Segmentation
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Report Metric
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Details
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Forecast Period
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2023 to 2030
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Base Year
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2022
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Historic Years
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2021 (Customizable to 2015-2020)
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Quantitative Units
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Revenue in USD Million, Volumes in Units, Pricing in USD
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Segments Covered
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Type (Fabry Disease, Tay-Sachs disease, and Others), Treatment (Enzyme Replacement Therapy, Stem Cell Transplantation, and Others), Route of Administration (Oral, Parenteral, and Others), End-Users (Hospitals, Specialty Clinics, Homecare, and Others), Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Pharmacy, and Others)
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Countries Covered
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U.S., Canada and Mexico, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific , Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa, Brazil, Argentina and Rest of South America
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Market Players Covered
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Novartis AG (Switzerland), Johnson & Johnson Private Limited (U.S.), Teva Pharmaceutical Industries Ltd. (Ireland), Merck & Co., Inc. (U.S.), Allergan (Ireland), Pfizer Inc. (U.S.), GlaxoSmithKline plc (U.K.), Sanofi (France), Merck KGaA (Germany), Abbott (U.S.), Boehringer Ingelheim International GmbH. (Germany), Takeda Pharmaceutical Company Limited (Japan), Amicus Therapeutics, Inc. (U.S.), Moderna, Inc. (U.S.),Greenovation Biotech GmbH (Germany), Biomarin (U.S.), JCR Pharmaceuticals Co., Ltd (Japan), ISU ABXIS (South Korea), Idorsia Pharmaceuticals Ltd (Switzerland), AVROBIO, Inc. (U.S.), Resverlogix Corp. (Canada), Enzyvant (U.S.), CHIESI Farmaceutici SpA (Italy)
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Market Opportunities
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Market Definition
Lysosomal or lysosomal storage disease is a rare hereditary metabolic disease. It is caused by faulty or mutant metabolism, which results in the lack or deficiency of enzymes, resulting in an abnormal material build-up in various body cells. If done before age two, bone marrow transplantation (BMT) successfully prevents progressive mental retardation in children with MPS IH (Hurler disease). Enzyme Replacement Therapy (ERT) is successful for those with Gaucher disease type I. Anemia and low platelet counts have improved, liver and spleen enlargement has decreased significantly, and skeletal abnormalities have improved. Individuals with Gaucher disease types II and III who get ERT also see improvements in these systemic signs.
Lysosomal Storage Disorder Drugs Market Dynamics
Drivers
- Prevalence and Awareness:
The increasing recognition and diagnosis of lysosomal storage disorders worldwide have driven the demand for effective treatment options. As awareness about these rare genetic disorders grows, more patients are being identified, leading to a greater need for drug development.
- Unmet Medical Need
Lysosomal storage disorders are a group of rare genetic diseases characterized by the deficiency of specific enzymes within lysosomes, resulting in the accumulation of undigested materials in cells. These disorders often lead to severe, progressive symptoms and can be life-threatening. Due to the limited treatment options available, there is a significant unmet medical need for effective therapies, driving the development of drugs in this field.
- Orphan Drug Incentives
Governments and regulatory agencies worldwide have implemented various incentives to promote the development of drugs for rare diseases, including lysosomal storage disorders. These incentives may include extended market exclusivity, tax credits, research grants, and accelerated regulatory pathways. Such incentives encourage pharmaceutical companies to invest in research and development efforts for these rare conditions.
- Advances in Biotechnology
The advancements in biotechnology and drug discovery techniques have significantly contributed to developing novel therapies for lysosomal storage disorders. Innovative approaches such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors have shown promising results in preclinical and clinical studies, fueling further investment in this field.
Opportunities
- Large Addressable Market
While individual LSDs are rare, there are over 50 known lysosomal storage disorders, each with unique genetic defects and clinical manifestations. Collectively, these disorders affect a substantial number of patients worldwide. The combined prevalence of LSDs makes for a sizable addressable market, presenting an opportunity for drug developers and manufacturers.
- Limited Treatment Options
Historically, treatment options for LSDs were limited, primarily focusing on symptomatic management. However, recent advancements in understanding the underlying biology of these disorders have led to the development of innovative therapies. The availability of disease-modifying treatments, such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors, has expanded the treatment landscape, providing better options for patients and driving market growth.
Restraints/Challenges
- Limited Patient Population
Lysosomal storage disorders are rare genetic diseases, and each specific disorder affects a relatively small number of patients. The limited patient population poses challenges for drug developers and manufacturers in achieving economies of scale and generating substantial revenue. The small market size may also deter some companies from investing in the research and development of LSD drugs.
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High Development Cost
Developing drugs for rare diseases, including LSDs, can be expensive and time-consuming. The cost of conducting clinical trials, obtaining regulatory approvals, and ensuring safety and efficacy can be substantial. The relatively small patient population and the complex nature of LSDs may result in higher per-patient costs, making the development of LSD drugs financially challenging for some companies.
This Hospice Market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, the impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the Hospice Market Contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.
Recent Development
- In January 2021, M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced that the U.S. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs
- In July 2019, Takeda Pharmaceutical Company launched an enzyme replacement therapy portfolio for lysosomal storage disorders (LSD) in India, expanding its range of rare disease therapies nationwide
Global Lysosomal Storage Disorder Drugs Scope
The lysosomal storage disorder drugs are segmented on the basis of treatment, indication, and end User. The growth amongst these segments will help you analyze meagre growth segments in the industries and provide the users with a valuable market overview and market insights to help them make strategic decisions for identifying core market applications.
Treatment
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Substrate Reduction Therapy
- Others
Indication
- Gaucher's Disease
- Fabry Disease
- Pompe’s Syndrome
- Mucopolysaccharidosis,
- Others
End user
- Hospitals,
- Clinics
- Others
Lysosomal Storage Disorder Drugs Regional Analysis/Insights
The lysosomal storage disorder drugs is analysed, and market size insights and trends are provided by country, treatment, indication, and end user as referenced above.
The countries covered in the lysosomal storage disorder drugs report are U.S., Canada and Mexico, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific , Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa, Brazil, Argentina and Rest of South America
North America dominates the lysosomal storage disorder drugs because of the strong base of healthcare facilities, the strong presence of major players in the market, the rising prevalence of lysosomal disease, and the rising number of research activities in this region.
Asia-Pacific is expected to witness significant growth during the forecast period of 2023 to 2030 due to the increase in government initiatives to promote awareness, rise in medical tourism, growing research activities in the region, availability of massive untapped markets, large population pool, and the growing demand for quality healthcare in the region.
The country section of the report also provides individual market-impacting factors and changes in regulation in the market domestically that impact the current and future trends of the market. Data points like down-stream and upstream value chain analysis, technical trends and porter's five forces analysis, case studies are some of the pointers used to forecast the market scenario for individual countries. Also, the presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.
Healthcare Infrastructure Growth Installed base and New Technology Penetration
The lysosomal storage disorder drugs also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for lysosomal storage disorder drugs , impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the lysosomal storage disorder drugs . The data is available for historic period 2010-2020.
Competitive Landscape and lysosomal storage disorder drugs Share Analysis
The lysosomal storage disorder drugs competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies' focus related to lysosomal storage disorder drugs .
Some of the major players operating in the lysosomal storage disorder drugs are:
- Novartis AG (Switzerland)
- Johnson & Johnson Private Limited (U.S.)
- Teva Pharmaceutical Industries Ltd. (Ireland)
- Merck & Co., Inc. (U.S.)
- Allergan (Ireland)
- Pfizer Inc. (U.S.)
- GlaxoSmithKline plc (U.K.)
- Sanofi (France)
- Merck KGaA (Germany)
- Abbott (U.S.)
- Boehringer Ingelheim International GmbH. (Germany)
- Takeda Pharmaceutical Company Limited (Japan)
- Amicus Therapeutics, Inc. (U.S.)
- Moderna, Inc. (U.S.)
- Greenovation Biotech GmbH (Germany)
- Biomarin (U.S.)
- JCR Pharmaceuticals Co., Ltd (Japan)
- ISU ABXIS (South Korea)
- Idorsia Pharmaceuticals Ltd (Switzerland)
- AVROBIO, Inc. (U.S.)
- Resverlogix Corp. (Canada)
- Enzyvant (U.S.)
- CHIESI Farmaceutici SpA (Italy)
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