US Lysosomal Storage Disease Therapeutics Market Analysis
US lysosomal storage disease therapeutics market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023-30. The rise in lysosomal diseases, greater public awareness of unusual lysosomal storage disorders, increased research and development for diagnostics, and increased drug development for treating lysosomal diseases are the main drivers impacting the market for lysosomal storage disease treatments. Major global players in this market are Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Therapeutics, Inc, Alexion Pharmaceuticals, Inc, Sigilon Therapeutics, Inc, and Orphazyme A/S are some of the companies that participate in this industry.
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US Lysosomal Storage Disease Therapeutics Market Analysis Summary
US Lysosomal Storage Disease Therapeutics Market is valued at around $3.61 Bn in 2022 and is projected to reach $5.09 Bn by 2030, exhibiting a CAGR of 4.4% during the forecast period 2023-2030.
Lysosomal enzyme shortage or dysfunction results in a set of uncommon inherited metabolic disorders known as lysosomal storage diseases. Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, aspartylglucosaminuria, Batten disease, cystinosis, and others are examples of lysosomal storage diseases. Currently, 11 lysosomal storage diseases can be treated using 23 orphan drugs. The prevalence of lysosomal storage disorders is increasing, people are becoming more aware of these diseases, and new medical technology is being developed to treat these ailments are all factors contributing to the market expansion. Major global players in this market are Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Therapeutics, Inc, Alexion Pharmaceuticals, Inc, Sigilon Therapeutics, Inc, and Orphazyme A/S are some of the companies that participate in this industry.
Market Dynamics
Market Growth Drivers
It is anticipated that throughout the course of the projection period, the global market for therapies for lysosomal storage diseases will expand due to the significant prevalence of LSDs. For instance, Fabry disease frequency in white, male populations ranged from 1:17,000 to 1:117,000, according to a National Center for Biotechnology Information report that was updated in January 2020. Between 1:6000 and 1:40,000 females and 1:1000 to 1:3000 males had atypical presentations, whereas between 1:22,000 and 140,000 males had the characteristic Fabry disease mutations. Additionally, it is hoped that increased public awareness of uncommon diseases will encourage market expansion. For instance, the European Organization for Rare Diseases celebrates Rare Disease Day on the last day of February each year.
Developments of US Lysosomal Storage Disease Therapeutics Market
Major industry companies are concentrating on putting M&A plans into practice in order to expand the scope of their product offering. For instance, Quest Diagnostics bought Blueprint Genetics in January 2020 to increase the range of its product offerings for uncommon and genetic disorders.
Major market players are concentrating on putting partnership ideas into practice in order to expand the scope of their portfolios. For instance, the University of Pennsylvania licensed Amicus Therapeutics, Inc. to use the Wilson Lab's Next Generation Gene Therapy Technologies for LSDs and other twelve uncommon illnesses in 2019.
Key players
Sanofi Genzyme Shire (Takeda Pharmaceutical Company Limited) BioMarin Pharmaceutical Inc. Pfizer Inc. Amicus Therapeutics, Inc. Regenxbio Inc. Ultragenyx Pharmaceutical Inc. Denali Therapeutics Inc. Avrobio, Inc. JCR Pharmaceuticals Co., Ltd.1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country
2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)
3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints
4. Competitive Landscape
4.1 Major Market Share
4.2 Key Company Profile (Check all Companies in the Summary Section)
4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)
5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment
6. Methodology and Scope
US Lysosomal Storage Disease Therapeutics Market Segmentation
By Treatment Therapy:
- Enzyme Replacement Therapy
- Velaglucerase Alfa
- Taliglucerase Alfa
- Agalsidase beta
- Laronidase
- Imiglucerase
- Other Enzyme Replacement Therapies
- Substrate Reduction Therapy
- Eliglustat
- Miglustat
- Other Substrate Reduction Therapies
By Application:
- Gaucher Disease
- Cystinosis
- Pompe Disease
- Fabry Disease
- Other Applications
By Sales Channel:
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
Methodology for Database Creation
Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.
Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.
How Do We Get It?
Our database is created and maintained through a combination of secondary and primary research methodologies.
1. Secondary Research
With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:
- Analyzing historical proprietary data collected from multiple projects.
- Regularly updating our existing data sets with new findings and trends.
- Ensuring data consistency and accuracy through rigorous validation processes.
With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:
- Searching through academic conferences, published research, citations, and social media platforms
- Collecting and compiling diverse data to build a comprehensive and detailed database
- Continuously updating our database with new information to ensure its relevance and accuracy
2. Primary Research
To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:
- Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
- Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
- Continuously refreshing our database to ensure that the information remains current and reliable.
- Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.
Combining Secondary and Primary Research
By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:
- Merging historical data from secondary research with real-time data from primary research.
- Conducting thorough data validation and cleansing to remove inconsistencies and errors.
- Organizing data into a structured format that is easily accessible and usable for various applications.
- Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.
Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.
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