The Gaucher and Pompe diseases enzyme replacement therapy (ERT) market is estimated to secure a valuation of USD 3.3 billion in 2023 and is expected to rise to USD 6.2 billion by 2033. The market is likely to capture a CAGR of 6.6% during the forecast period. The following factor driving the market is
High Cost of Treatment May Hinder Market Growth
Medicine and enzyme replacement therapy for Gaucher and Pompe disease may improve patients' conditions, although the market has a few restraining factors that may cause a decline in growth.
The increasing treatment cost of Pompe disease and its side effects may limit the market growth. The lack of skilled experts and medical workers and the adoption of alternative treatment options also decline the market growth.
While ERT has improved the prognosis in both infantile and later-onset Pompe and Gaucher diseases, the presence of the blood-brain barrier and pathological preconditions obstructs the effective uptake of ERT. As a result, efforts are being made to develop more precise treatment approaches. However, a shortage of skilled health professionals for this therapy and poor reimbursement regulations in emerging nations constrain market expansion.
Challenges Faced by Patients During COVID-19 Crisis Globally
Through distribution channels such as retail pharmacies, online pharmacies, hospital pharmacies, and speciality treatment pharmacies, the market is expanding around the globe. During the COVID-19 crisis, the healthcare sector was temporarily closed due to lockdowns, due to which patients suffering from chronic diseases faced several challenges with their regular medical checkups.
The distribution channels and suppliers stop their services due to social distancing. In the global market, the lack of medicine, doses, and shortage of beds and equipment decline the market size. The high-risk patients of lysosomal diseases due to direct involvement in respiratory, cardiac, and rental issues faced challenges. The operations, checkups, and appointments got postponed, and tragic deaths from the COVID-19 virus shook patients' minds due to fear.
| Attributes | Details |
|---|---|
| Market CAGR (2023 to 2033) | 6.6% |
| Market Valuation (2023) | USD 3.3 billion |
| Market Valuation (2033) | USD 6.2 billion |
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According to Future Market Insights, the global Gaucher and Pompe diseases enzyme replacement therapy (ERT) market registered a healthy 6.6% CAGR during the forecast period. Historically, the market secured a CAGR of 7.4% between 2017 and 2022.
In enzyme replacement therapy, patients are administered various types of enzymes to overcome enzyme shortages/malfunctions. Increased prevalence of rare, chronic, and inherited diseases such as lysosomal storage diseases and genetic disorders is leading to the need for effective treatment options.
Infusions of an enzyme isolated from human or animal tissue or blood, or synthesized using innovative recombinant DNA techniques, replace the missing enzyme. A longer half-life of the drug, more potent action, resistance to degradation, or targeting a specific organ, tissue, or cell type are all possible modifications to an enzyme.
The body can complete the processes impeded by the deficiency by getting these enzyme replacements. Enzyme replacement therapies are designed to raise the concentration of enzymes, not to change the disease's underlying cause. ERT has been available for various lysosomal storage disorders, including several mucopolysaccharidoses (MPS).
Short Term (2023 to 2026): Changing lifestyles, healthcare infrastructure, eating habits, and a growing chronic population are increasing the adoption of Gaucher and Pompe diseases enzymes replacement therapy (ERT) during the time frame. Additionally, manufacturers in industrialized countries are focused on developing advanced technology for Gaucher disease therapy, which may further upsurge the market globally.
Medium Term (2026 to 2029): The discovery of beta-glucosidase deficiency, increasing diagnosis and treatment availability, and innovation in enzyme replacement therapy are expected to propel market growth. Manufacturers grappling with improving quality and developing products to satisfy patients' requirements may flourish the market opportunities in this period.
Long Term (2029 to 2033): Manufacturers are diving into research and development activities during the projected period to innovate high-quality therapy treatments without patient side effects. New market entrants with considerable investments in enzyme deficiency are advancing the market size.
The market is estimated to reach USD 6.2 billion during the forecast period. Historically, the market stood at USD 3.1 billion from 2017 to 2022.
| Segment | Therapeutic Condition |
|---|---|
| Top Category | Gaucher Disease |
| Historic CAGR | 7.8% |
| Forecast CAGR | 7.0% |
| Segment | Distribution Channel |
|---|---|
| Top Category | Specialty Treatment Pharmacies |
| Historic CAGR | 7.3% |
| Forecast CAGR | 6.6% |
Based on distribution channels, specialty treatment pharmacies are anticipated to dominate the global market share by securing a CAGR of 6.6% by 2033. The availability of treatment and patient preferences are growing in specialty treatment pharmacies may drive the market growth. Historically, specialty treatment pharmacies secured a CAGR of 7.3% between 2017 and 2022.
The growing popularity of retail and online pharmacies is due to the rising patients' demand for home-based ERT treatments. After the pandemic, online pharmacies rapidly grew, accounting for around 24.4% of the global market.
Hospital pharmacies are growing significantly by capturing a relevant CAGR in the global market by 2033. Emerging economies, advanced healthcare facilities, skilled professionals, and infusion therapy are advancing hospital pharmacies.
Principal Consultant
| Country | United States |
|---|---|
| Historic CAGR | 7.0% |
| Forecast CAGR | 6.1% |
| Valuation 2033 | USD 1,329.3 million |
| Country | China |
|---|---|
| Historic CAGR | 8.0% |
| Forecast CAGR | 7.1% |
| Valuation 2033 | USD 335.8 million |
| Country | Canada |
|---|---|
| Historic CAGR | 7.4% |
| Forecast CAGR | 6.9% |
| Valuation 2033 | USD 159.5 million |
| Country | Germany |
|---|---|
| Historic CAGR | 7.0% |
| Forecast CAGR | 6.1% |
| Valuation 2033 | USD 465.4 million |
| Country | United Kingdom |
|---|---|
| Historic CAGR | 7.8% |
| Forecast CAGR | 6.9% |
| Valuation 2033 | USD 477.9 billion |
The United States is anticipated to dominate the global market by capturing a CAGR of 6.1% with a valuation of USD 1,329.3 million by 2033. The rising urbanization, emerging economies, and developing healthcare infrastructure propel the United States Gaucher and Pompe diseases enzyme replacement therapy (ERT) market.
The growing technological advanced in healthcare equipment adoption of AI and IoT for diagnosis are fueling the market opportunities in the country. Healthcare providers research medicine by adopting the latest technologies. Increasing huge investment, urbanization, and new product launches are advancing the market size in the country. In the historical period, the United States secured a CAGR of 7.0% between 2017 and 2022.
China is anticipated to capture a CAGR of 7.1% with a valuation of USD 335.8 million by 2033. The increasingly advanced healthcare infrastructure, medical facilities, and research and development activities fuel China's Gaucher and Pompe diseases enzyme replacement therapy (ERT) market. The country's innovation of enzyme replacement therapy is expanding its huge revenue.
The growing generic population, uncommon disorders, and Pompe disease treatment drive the market size. China captured a CAGR of 8.0% in the historical period between 2017 and 2022.
The United States is capturing a CAGR of 6.9% with a valuation of USD 477.9 million during the forecast period. The increasing treatment modalities, hospital pharmacies, and therapy products fuel the market size. The strong presence of key players, increasing lysosomal disorder diseases, and Fabry disease fuel the market size.
The growing infusion facilities, healthcare infrastructure, government initiatives, and cutting-edge medical treatment drive the United Kingdom Gaucher and Pompe diseases enzyme replacement therapy (ERT) market. Healthcare manufacturers are developing budget-friendly therapy for low and middle-class patients to satisfy their requirements in the country. In the historical period, the United Kingdom captured a CAGR of 7.8% between 2017 and 2022.
Competitive Landscape
The essential players enact a significant role in the global market. Various healthcare providers and the pharmaceuticals sector focus on providing advanced equipment to satisfy patients' requirements. The prominent players invest a huge amount in research and development activities to acquire maximum output in the global market.
These players adopt marketing strategies, including partnerships, acquisitions, collaborations, product launches, agreements, and mergers. These players collect maximum revenue in the global market through distribution channels and suppliers. Launching innovative products and various therapeutic ailments are fueling the market size at another level.
A strong focus on acquiring regulatory approval further allows them to meet the increased need for treating lysosomal storage disorders across the globe.
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| Attribute | Details |
|---|---|
| Forecast Period | 2023 to 2033 |
| Historical Data Available for | 2017 to 2022 |
| Market Analysis | USD billion for Value |
| Key Countries Covered | United States, Canada, Germany, United Kingdom, France, Italy, Spain, Russia, China, Japan, South Korea, India, Thailand, Malaysia, Indonesia, Australia, New Zealand, GCC Countries, Türkiye, Northern Africa, and South Africa |
| Key Segments Covered | Therapeutic Condition, Route of Administration, Distribution Channel, Region |
| Key Companies Profiled | Alexion Pharmaceuticals, Inc.; Pfizer; BioMarin Pharmaceuticals Inc; Ultragenyx Pharmaceutical Inc.; Janssen Pharmaceuticals, Inc (J&J); Sigma-Tau Pharmaceuticals Inc; AbbVie Inc.; Sanofi S.A.; Shire Plc; Merck KGaA; Takeda Pharmaceuticals |
| Report Coverage | Market Forecast, Company Share Analysis, Competition Intelligence, DROT Analysis, Market Dynamics and Challenges, and Strategic Growth Initiatives |
| Customization & Pricing | Available upon Request |
China, India, and Japan dominate the Asian market.
The market is valued at USD 3.3 billion in 2023.
Sanofi, Genzyme Corporation, and Shire are key market players.
Japan, China, and India dominate the Asian market.
The market is estimated to reach USD 6.2 billion by 2033.
North America is projected to emerge as a lucrative market.
1. Executive Summary | Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market 2. Market Overview 3. Market Background 4. Global Market Analysis 2018 to 2022 and Forecast, 2023 to 2033 5. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Disease 5.1. Gaucher Disease 5.1.1. Type 1 (Non-neuropathic) 5.1.2. Type 2 (Acute Infantile Neuronopathic) 5.1.3. Type 3 Gaucher Disease (Chronic Neuronopathic) 5.1.4. Perinatal Lethal Gaucher Disease 5.1.5. Cardiovascular Gaucher Disease 5.2. Pompe Disease 5.2.1. Classic Infantile-onset 5.2.2. Non-classic Infantile-onset 5.2.3. Late-onset 6. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Route of Administration 6.1. Oral 6.2. Parenteral 7. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Distribution Channel 7.1. Hospital Pharmacies 7.2. Specialty Treatment Pharmacies 7.3. Retail Pharmacies 7.4. Online Pharmacies 8. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Region 8.1. North America 8.2. Latin America 8.3. Europe 8.4. South Asia 8.5. East Asia 8.6. Oceania 8.7. MEA 9. North America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 10. Latin America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 11. Europe Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 12. South Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 13. East Asia Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 14. Oceania Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 15. MEA Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 16. Key Countries Market Analysis 17. Market Structure Analysis 18. Competition Analysis 18.1. Pfizer 18.2. Alexion Pharmaceuticals, Inc. 18.3. BioMarin Pharmaceutical Inc. 18.4. Ultragenyx Pharmaceutical Inc. 18.5. Janssen Pharmaceuticals, Inc. 18.6. Sigma-Tau Pharmaceuticals Inc. 18.7. AbbVie Inc. 18.8. Sanofi S.A. 18.9. Shire Plc 18.10. Merck KGaA 18.11. Takeda Pharmaceuticals 19. Assumptions & Acronyms Used 20. Research Methodology
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Gaucher and Pompe Diseases Enzyme Replacement Therapy (ERT) Market
