Image

Global Lysosomal Storage Disorder Drugs Market – Industry Trends and Forecast to 2030

Pharmaceutical

Image

Global Lysosomal Storage Disorder Drugs Market – Industry Trends and Forecast to 2030

  • Pharmaceutical
  • Upcoming Report
  • Jul 2023
  • Global
  • 350 Pages
  • No of Tables: 220
  • No of Figures: 60

Global Lysosomal Storage Disorder Drugs Market – Industry Trends and Forecast to 2030

Market Size in USD Billion

CAGR :  % Diagram

Diagram Forecast Period 2022–2030
Diagram Market Size (Base Year) USD 9,738.63 USD Million
Diagram Market Size (Forecast Year) USD 21,646.92 USD Million
Diagram CAGR %

Major Markets Players

  • Dummy1
  • Dummy2
  • Dummy3
  • Dummy4
  • Dummy5

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

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

Report Metric

Details

Forecast Period

2023 to 2030

Base Year

2022

Historic Years

2021 (Customizable to 2015-2020)

Quantitative Units

Revenue in USD Million, Volumes in Units, Pricing in USD

Segments Covered

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)

Countries Covered

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

Market Players Covered

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)

Market Opportunities

  • Growing number of research and development activities
  • Technological advancement

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.

  • 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) 


SKU-

Please fill in the below form for detailed Table of Content

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Please fill in the below form for detailed List of Table

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Please fill in the below form for detailed List of Figure

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Please fill in the below form for Infographics

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Research Methodology:

Data collection and base year analysis are done using data collection modules with large sample sizes. The stage includes obtaining market information or related data through various sources and strategies. It includes examining and planning all the data acquired from the past in advance. It likewise envelops the examination of information inconsistencies seen across different information sources. The market data is analysed and estimated using market statistical and coherent models. Also, market share analysis and key trend analysis are the major success factors in the market report. To know more, please request an analyst call or drop down your inquiry.

The key research methodology used by DBMR research team is data triangulation which involves data mining, analysis of the impact of data variables on the market and primary (industry expert) validation. Data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Patent Analysis, Pricing Analysis, Company Market Share Analysis, Standards of Measurement, Global versus Regional and Vendor Share Analysis. To know more about the research methodology, drop in an inquiry to speak to our industry experts.

Please fill in the below form for Research Methodology

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Customization Available:

Data Bridge Market Research is a leader in advanced formative research. We take pride in servicing our existing and new customers with data and analysis that match and suits their goal. The report can be customized to include price trend analysis of target brands understanding the market for additional countries (ask for the list of countries), clinical trial results data, literature review, refurbished market and product base analysis. Market analysis of target competitors can be analyzed from technology-based analysis to market portfolio strategies. We can add as many competitors that you require data about in the format and data style you are looking for. Our team of analysts can also provide you data in crude raw excel files pivot tables (Fact book) or can assist you in creating presentations from the data sets available in the report.

Please fill in the below form for Available Customization

By clicking the "Submit" button, you are agreeing to the Data Bridge Market Research Privacy Policy and Terms and Conditions

Get online access to the report on the World's First Market Intelligence Cloud

  • Interactive Data Analysis Dashboard
  • Company Analysis Dashboard for high growth potential opportunities
  • Research Analyst Access for customization & queries
  • Competitor Analysis with Interactive dashboard
  • Latest News, Updates & Trend analysis
  • Harness the Power of Benchmark Analysis for Comprehensive Competitor Tracking
Request for Demo

FREQUENTLY ASK QUESTIONS

The Lysosomal Storage Disorder Drugs Market will be worth USD 21646.92 million in 2030.
The Lysosomal Storage Disorder Drugs Market Growth Rate is 10.50% during the forecast period.
The Prevalence and Awareness, Advances in Biotechnology and Orphan Drug Incentives are the Growth Drivers of the Lysosomal Storage Disorder Drugs Market.
The category, grade, source, application, and technical type are the factors on which the Lysosomal Storage Disorder Drugs Market research is based.
The Takeda Pharmaceutical Company launched an enzyme replacement therapy portfolio for lysosomal storage disorders (LSD) in India, expanding its range of rare disease therapies nationwide are the latest developments in the Lysosomal Storage Disorder Drugs Market.
Free Sample Report

CHOOSE LICENCE TYPE

  • 7000.00
  • 4800.00
  • 3000.00
  • 8000.00
  • 12000.00

Why Choose Us

Industry Coverage

DBMR works across the globe in multiple industries which equip us with knowledge across verticals and provide our clients with insights not only from their industry but how other industries will impact their ecosystem.

Regional Coverage

Coverage of Data Bridge is not restricted to developed or emerging economies. We work across the globe covering the largest array of countries where no other market research or business consulting firm has ever conducted research; creating growth opportunities for our clients in areas which are still unknown.

Technology Coverage

In today’s world, technology drives the market sentiment, so our vision is to provide our clients insights not only for developed technologies but upcoming and disrupting technological changes throughout the product lifecycle by enabling them with unforeseen opportunities in the market which will create disruption in their industry. This leads to innovation and our clients to come out as winners.

Goal Oriented Solutions

DBMR goal is to help our clients achieve their goals through our solutions; hence we formatively create the most appropriate solutions for our client needs, saving time and efforts for them to drive their grand strategies.

Unparallel Analyst Support

Our analysts take pride in our clients’ success. Unlike others, we believe in working along our clients to achieve their goals with 24 hours analyst support determining the correct needs and inspire innovation through service.

Banner

Client Testimonials