PUBLISHER: The Business Research Company | PRODUCT CODE: 1668370
PUBLISHER: The Business Research Company | PRODUCT CODE: 1668370
Lysosomal diseases (LSDs) are characterized by the accumulation of substrates within cells of various organs due to impaired lysosomal function. This accumulation of materials in the lysosomes results in damage to various tissues and organs, giving rise to a diverse range of symptoms that can impact different parts of the body.
The primary types of treatment for lysosomal diseases encompass conditions such as mucopolysaccharidosis, Pompe's syndrome, Fabry disease, Gaucher's disease, and others. Mucopolysaccharidosis type I (MPS I), for example, is an LSD caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), and it is treated through enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT). Therapies for these diseases include substrate reduction therapy, stem cell therapy, enzyme replacement therapy, and other treatments administered through various routes, such as oral, parenteral, and others. These treatments are administered in settings such as hospitals, specialty clinics, home care, and other end-user environments.
The lysosomal disease treatment market research report is one of a series of new reports from The Business Research Company that provides lysosomal disease treatment market statistics, including lysosomal disease treatment industry global market size, regional shares, competitors with a lysosomal disease treatment market share, detailed lysosomal disease treatment market segments, market trends and opportunities, and any further data you may need to thrive in the lysosomal disease treatment industry. This lysosomal disease treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The lysosomal disease treatment market size has grown strongly in recent years. It will grow from$9.22 billion in 2024 to $9.88 billion in 2025 at a compound annual growth rate (CAGR) of 7.2%. The growth in the historic period can be attributed to advancements in therapeutic approaches, improved understanding and awareness, rise in clinical trials and research efforts, enhancements in diagnostic tools, development of newborn screening programs
The lysosomal disease treatment market size is expected to see strong growth in the next few years. It will grow to $13.7 billion in 2029 at a compound annual growth rate (CAGR) of 8.5%. The growth in the forecast period can be attributed to gene therapy advancements, continued developments in enzyme replacement therapy (ERT), expansion of new therapeutic modalities, enhanced screening and early diagnosis programs, investments in research and development. Major trends in the forecast period include advancements in enzyme replacement therapy (ERT), gene therapy innovations, chaperone therapy development, substrate reduction therapy (SRT), expansion of newborn screening programs.
The increasing number of diagnostic activities is expected to drive the growth of the lysosomal disease treatment market. Diagnostic activities refer to systematic processes and tests used to identify and analyze problems, conditions, or diseases in individuals or systems, often involving data collection and interpretation to guide decision-making and treatment strategies. Factors fueling the growth of diagnostic activities include advancements in medical technology, a rising prevalence of chronic diseases, growing demand for preventive healthcare, heightened awareness and education regarding health issues, and supportive government policies and funding for healthcare initiatives. As diagnostic methods become more advanced, healthcare providers can identify lysosomal diseases more accurately, leading to increased investment in targeted therapies and treatments. For instance, in May 2023, the National Health Service (NHS), a UK-based publicly funded healthcare system, reported that 2,302,400 diagnostic tests were conducted in March 2023, an increase of 223,100 tests compared to March 2022. Additionally, by the end of March 2023, the number of patients waiting for a key diagnostic test reached 1,628,300, an increase of 59,400 from March 2022. Thus, the growth in diagnostic activities is driving the expansion of the lysosomal disease treatment market.
The growth of the lysosomal disease treatment market is further fueled by increased investments in healthcare infrastructure. Investments directed towards the development, maintenance, and enhancement of healthcare facilities and services contribute significantly to healthcare efficiency and patient outcomes. Notably, Russia's federal project for modernizing primary healthcare is set to allocate substantial funds, amounting to 88.8 billion rubles ($910 million) in 2023 and 550 billion rubles ($5.7 billion) for 2023-2025. This underscores the positive impact of growing investments in healthcare infrastructure on the lysosomal disease treatment market.
Major companies operating in the lysosomal disease treatment market are focused on developing innovative solutions, such as dual-component therapy designed to enhance enzyme replacement and improve muscle function in patients. A dual-component therapy is a treatment approach that combines two complementary agents or drugs to work together, enhancing overall therapeutic effectiveness for a specific condition. For instance, in September 2023, Amicus Therapeutics, a US-based biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65 mg capsules. Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) are a dual-component therapy aimed at treating late-onset Pompe disease, a severe lysosomal disorder characterized by glycogen accumulation due to enzyme deficiency. This combination enhances enzyme uptake in muscle cells while stabilizing the enzyme in circulation, effectively improving musculoskeletal strength and respiratory function in patients who have not responded to existing therapies. Clinical trials indicate that this innovative approach may offer significant benefits, addressing unmet needs in the management of this debilitating condition.
Major players in the lysosomal disease treatment market are strategically focusing on research and technology advancements to solidify their market presence. Research and technological progress signify continuous improvements and breakthroughs in scientific investigation and capabilities. For instance, in September 2023, Amicus Therapeutics, a US-based biopharmaceutical company, received FDA approval for Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) 65mg capsules for the treatment of adult patients with late-onset Pompe disease (LOPD). This two-component therapy, comprising cipaglucosidase alfa-atga and miglustat, showcases innovative advancements in lysosomal disease treatment.
In March 2024, CENTOGENE, a Germany-based company specializing in rare disease diagnostics and multiomic data insights, partnered with Takeda to expedite treatments for rare genetic diseases. Through this extended partnership, CENTOGENE aims to enhance its collaboration with Takeda by utilizing its proprietary multiomic technology platform to accelerate the discovery and development of innovative treatments for rare genetic disorders. Takeda, a Japan-based biopharmaceutical company, focuses on researching and developing highly specialized therapies for rare diseases and other therapeutic areas.
Major companies operating in the lysosomal disease treatment market include Eli Lilly and Company, Takeda Pharmaceutical Company Limited, Sanofi SA, Pfizer Inc., Novartis International AG, Johnson & Johnson Services Inc., Alexion Pharmaceuticals Inc., Amicus Therapeutics Inc., Actelion Pharmaceuticals Ltd., Sigilon Therapeutics Inc., BioMarin Pharmaceutical Inc., Merck & Co Inc., AstraZeneca PLC, Astellas Pharma Inc., Horizon Therapeutics plc, Leadient Biosciences Inc., Orphazyme A/S, Recordati Industria Chimica e Farmaceutica S.p.A., Valerion Therapeutics Llc, Viatris Inc., Chiese Farmaceutici SpA, Genzyme Corporation, Ultragenyx Pharmaceutical Inc., Sangamo Therapeutics Inc., Avrobio Inc., Axovant Gene Therapies Ltd., Regenxbio Inc., Krystal Biotech Inc., Homology Medicines Inc., Orchard Therapeutics plc, Sarepta Therapeutics Inc., Solid Biosciences Inc.
North America was the largest region in the lysosomal disease treatment market in 2024. Europe is expected to be the fastest-growing region in the global lysosomal disease treatment market report during the forecast period. The regions covered in the lysosomal disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa
The countries covered in the lysosomal disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain
The lysosomal disease treatment market includes revenues earned by entities by offering lysosomal disease treatment services for inherited lysosomal disorders such as neuronal ceroid lipofuscinoses, Danon disease, and Wolman disease, and diagnosis of the disease through medication and therapies. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included. The lysosomal disease treatment market consists of sales of medicines and services such as Bone marrow transplantation, the use of molecular chaperones, and Gene therapy. Values in this market are 'factory gate' values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
Lysosomal Disease Treatment Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses on lysosomal disease treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
Where is the largest and fastest growing market for lysosomal disease treatment ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The lysosomal disease treatment market global report from the Business Research Company answers all these questions and many more.
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.
The forecasts are made after considering the major factors currently impacting the market. These include the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.