Fabry Disease Treatment Global Market Report 2024

Fabry disease stems from a deficiency in alpha-galactosidase A, an enzyme crucial for breaking down globotriaosylceramide (Gb3 or GL-3) fat. This results in Gb3 accumulation within cells, prompting symptoms such as pain, kidney issues, heart complications, and strokes. Treatment aims to curtail Gb3 buildup, manage symptoms, slow organ damage, and enhance patients' quality of life.

The fabry disease treatment landscape includes substrate reduction therapy, enzyme replacement therapy, chaperone interventions, among others. Substrate reduction therapy (SRT) targets the reduction of specific substances in the body linked to fabry disease. These therapies can be administered orally or intravenously and cater to varying disease severities such as classic and late-onset fabry disease. Distribution channels encompass hospital, retail, and online pharmacies.

The fabry disease treatment market research report is one of a series of new reports that provides fabry disease treatment market statistics, including the fabry disease treatment industry global market size, regional shares, competitors with fabry disease treatment market share, detailed fabry disease treatment market segments, market trends, and opportunities, and any further data you may need to thrive in the fabry disease treatment industry. These fabry disease treatment market research reports deliver a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The fabry disease treatment market size has grown strongly in recent years. It will grow from $1.92 billion in 2023 to $2.09 billion in 2024 at a compound annual growth rate (CAGR) of 8.8%. Historical growth factors include heightened awareness and diagnosis rates, greater adoption of enzyme replacement and chaperone therapies, expanded healthcare infrastructure, increased investment in rare disease research, a growing population, and regulatory incentives promoting orphan drug development.

The fabry disease treatment market size is expected to see strong growth in the next few years. It will grow to $2.94 billion in 2028 at a compound annual growth rate (CAGR) of 8.9%. Forecasted growth drivers encompass the disease's increasing prevalence, heightened awareness and diagnosis rates, augmented healthcare infrastructure, amplified investment in rare disease research, rising patient awareness, and broader reimbursement policies. Anticipated trends include advancements in gene therapy, enzyme replacement therapies, diagnostic techniques, novel therapies, and the integration of digital health tech for enhanced patient care.

The growth of the Fabry disease treatment market is expected to be driven by the increasing prevalence of renal diseases. Renal diseases, or kidney diseases, encompass conditions that affect kidney function and are on the rise due to genetic factors, lifestyle choices, environmental influences, and underlying health issues. Fabry disease, specifically caused by the buildup of globotriaosylceramide (Gb3) in kidney cells, results in kidney damage and dysfunction. This emphasizes the urgent need for early detection and intervention to prevent kidney-related complications. For instance, recent data from the Australian Bureau of Statistics revealed a 0.2% increase in kidney disease cases over the past decade, with 246,200 people (1% of the population) affected in 2022, highlighting the escalating prevalence of renal diseases and their impact on the fabry disease treatment market.

Key players in the Fabry disease treatment market are actively developing plant cell-based therapies to enhance treatment efficacy, reduce costs, and improve patient outcomes through innovative and sustainable methods. Plant cell-based treatments for fabry disease involve using plant cells to produce deficient enzymes found in individuals with fabry disease. These plant-derived enzymes serve as a therapeutic option to replace the missing or defective enzymes in the body, aiming to alleviate symptoms and enhance patients' overall health. For example, ELFABRIO, an enzyme replacement therapy developed by Chiesi Global Rare Diseases and Protalix BioTherapeutics Inc., was approved in May 2023. ELFABRIO utilizes recombinant human α-Galactosidase-A enzyme produced in plant-cell culture and modified with PEGylation to extend its half-life significantly. This innovative treatment approach reduces treatment frequency, increases convenience, and improves symptom management, ultimately enhancing the quality of life for fabry disease patients.

In March 2024, CENTOGENE extended its partnership with Takeda Pharmaceutical Company Limited, a leader in developing therapies for lysosomal storage disorders (LSDs) such as fabry disease. This collaboration ensures continued access to diagnostic testing for patients globally, improving patient access to rapid and accurate diagnostics for LSDs, including fabry disease.

Major companies operating in the fabry disease treatment market are Sanofi S.A., GSK plc, Takeda Pharmaceutical Company Limited, CHIESI Farmaceutici S.p.A., Ultragenyx Pharmaceutical Inc., Amicus Therapeutics Inc., JCR Pharmaceuticals Co. Ltd., Sangamo Therapeutics Inc., Idorsia Pharmaceuticals Ltd., Centogene N.V., Protalix BioTherapeutics, Avrobio Inc., ISU Abxis, Synageva BioPharma Corp., Greenovation Biotech GmbH, CANbridge Pharmaceuticals, Freeline, 4D Molecular Therapeutics, Abeona Therapeutics Inc., GC Biopharma Corporation.

North America was the largest region in the fabry disease treatment market in 2023. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the fabry disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

The countries covered in the fabry disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The fabry disease treatment market consists of revenues earned by entities by providing services such as diagnosis and screening, enzyme replacement therapy, gene therapy, disease management programs, and patient education and support. The market value includes the value of related goods sold by the service provider or included within the service offering. The Fabry disease treatment market also includes sales of Agalsidase alfa, Agalsidase beta, and Migalastat. 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.

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