Lysosomal Disease Treatments Market: Introduction
Lysosomal storage disorders cause several diseases. Lysosomal storage diseases (LSDs) are a large group of disorders caused by a deficiency of specific enzymes responsible for the degradation of substances present in lysosomes. They affect the lysosome -- a structure in cells that breaks down substances, such as proteins, carbohydrates, and old cell parts so the body can recycle them. The purpose of substrate reduction therapy is to down regulate the formation of the lysosomal substance to a rate at which the residual enzyme activity can catabolize the stored and de novo produced lysosomal substrate. Chemical chaperone therapy is based on small molecules able to bind and stabilize the misfolded enzymes.
Clinical signs characterizing different diseases depend on the quantity and type of accumulated substance; in general, the disease is named after the type of undegraded substrate. Most LSDs have an autosomal recessive inheritance pattern. Mucopolysaccharidosis type II, Fabry disease, and Danon disease are X-linked. Different therapeutic approaches were used in the past to address the underlying causes of the diseases: infusion of plasma or plasma fractions, intravenous injection of exogenous enzymes extracted from human tissues, infusion of leukocytes and implantation of skin fibroblasts or amniotic cells. People with these disorders are missing important enzymes (proteins that speed up reactions in the body). Without the enzymes, the lysosome is not able to break down these substances.
Get a Glimpse of the In-Depth Analysis through our Comprehensive Lysosomal Disease Treatments Market Brochure
Key Drivers of Global Lysosomal Disease Treatments Market
Demand for orphan drug status
- Current economic situation, along with growing generic competition has shifted the focus of pharmaceutical companies from essential medicines to a new business model - niche busters, also called orphan drugs
- Orphan drugs may help pharma companies to reduce the impact of revenue loss caused by patent expiries of blockbuster drugs
- Rare diseases in small patient population, thus “orphaned” by the pharmaceutical industry having but a few approved drug treatment options available are called orphan diseases
- Orphan diseases are often so rare that a physician may observe only one case a year or less. Academic physician-scientists have tried to fill this therapy vacuum by working on developing orphan drugs.
North America to Hold Major Share of Global Lysosomal Disease Treatments Market
- North America is expected to hold a major share of the global lysosomal disease treatments market during the forecast period. The market in North America is estimated to expand at a rapid pace in the near future, primarily due to structured reimbursement policies, increase in prevalence of obesity & cardiovascular diseases, developed health care infrastructure, and continuous research activities in developing advanced technologies in North America.
- Lysosomal disease treatments market in Asia Pacific is projected to expand at a rapid pace during the forecast period. This can be attributed to growing trend of preventive health care measures.
Expanding Operations in Future? To Get the Perfect Launch Ask for a Custom Lysosomal Disease Treatments Market Report
Key Players Operating in Global Market
The global lysosomal disease treatments market is highly concentrated due to the presence of a few key players. Several manufacturers hold major share of the market in their respective regions. Leading players operating in the global lysosomal disease treatments market are:
- Shire PLC
- Pfizer, Inc.
- BioMarin Pharmaceutical Inc.
- Actelion Ltd.
- Raptor Pharmaceutical Corp.
- Protalix Biotherapeutics Inc.
- Amicus Therapeutics, Inc.
Global Lysosomal Disease Treatments Market: Research Scope
Global Lysosomal Disease Treatments Market, by Indication
- Aucher's Diseases
- Fabry Diseases
- Pompe’s Syndrome
Global Lysosomal Disease Treatments Market, by Type of Therapy
- Enzyme Replacement Therapy
- Stem Cell Therapy
- Substrate Reduction Therapy
Global Lysosomal Disease Treatments Market, by End-user
Global Lysosomal Disease Treatments Market, by Region
- North America
- Asia Pacific
- Australia & New Zealand
- Rest of Asia Pacific
- Latin America
- Rest of Latin America
- Middle East & Africa
- South Africa
- Rest of Middle East & Africa
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
2. Insights and Tools
The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
3. Actionable Results
The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.