Lysosomal Disease Treatment Global Market Report 2024

The lysosomal disease treatment market has grown strongly in recent years. It will grow from $8.69 billion in 2023 to $9.33 billion in 2024 at a compound annual growth rate (CAGR) of 7.3%. The expansion observed in the historical period can be attributed to advancements in therapeutic approaches, an improved understanding and awareness of lysosomal diseases, an increase in clinical trials and research efforts, enhancements in diagnostic tools, and the development of newborn screening programs.

The lysosomal disease treatment market is expected to see strong growth in the next few years. It will grow to $12.58 billion in 2028 at a compound annual growth rate (CAGR) of 7.8%. The anticipated growth in the forecast period can be attributed to advancements in gene therapy, ongoing developments in enzyme replacement therapy (ERT), the expansion of new therapeutic modalities, improved screening and early diagnosis programs, and increased investments in research and development. Major trends in the forecast period encompass innovations in enzyme replacement therapy (ERT), developments in gene therapy, the evolution of chaperone therapy, the progress of substrate reduction therapy (SRT), and the expansion of newborn screening programs.

The anticipated increase in the prevalence of lysosomal disorders is set to drive the growth of the lysosomal disease treatment market. Lysosomal diseases, characterized by rare genetic disorders leading to enzyme deficiencies and abnormal material accumulation in the body, necessitate treatment involving the degradation of stored materials within lysosomes. Exogenous enzymes synthesized externally are infused into the bloodstream to address these diseases. A study published in The Lancet in December 2021 revealed a combined prevalence of 1 per 4,800 live births for lysosomal storage disorders in Australia. The incidence involved 766 confirmed diagnoses across 38 different disorders, indicating a prevalence of LSD in adulthood more than in childhood. This rise in lysosomal disorders is a key factor propelling the growth of the home healthcare equipment 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.

A notable trend in the lysosomal disease treatment market is the collaborative innovation for novel disease treatments. Companies within the market are forging collaborations to advance treatment solutions, employing innovative technologies to maintain their market position. An example is the collaboration between Eli Lilly, a US-based pharmaceutical company, and Lycia Therapeutics, Inc., a US-based biotechnology company, aiming to develop and commercialize targeted therapeutics using Lycia's lysosomal targeting chimera (LYTAC) protein degradation technology. Such partnerships align with the strategy of leveraging innovative technology to address challenging disease areas, including lysosomal disorders.

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 July 2021, AstraZeneca, a UK-based pharmaceutical and biotechnology company, completed the acquiring Alexion Pharmaceuticals, Inc. (Alexion) for $39 billion. This acquisition aimed to bolster AstraZeneca's position in immunology, complementing its existing capabilities in oncology, respiratory, and cardiovascular disease. Alexion Pharmaceuticals, a US-based company, is renowned for developing treatments for rare diseases, including lysosomal disorders.

Major companies operating in the lysosomal disease treatment market report are 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, Arena Pharmaceuticals Inc., 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 2023. 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 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 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 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.


This product will be delivered within 3-5 business days.