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Lysosomal storage diseases (LSDs) are a group of over 50 disorders, each involving a different lysosomal enzyme. According to the National Organization for Rare Disorders, LSDs are believed to have a frequency of one in every 50,000 live births. Most LSDs are caused due to autosomal recessive inheritance leading to genetic mutations resulting in deficiency of lysosomal enzymes.

The global lysosomal storage diseases therapeutics market is estimated to account for US$ 7,217.9 Mn in terms of value in 2019 and is expected to reach US$ 15,734.5 Mn by the end of 2027.

Global Lysosomal Storage Diseases Therapeutics Market: Drivers

Favorable regulations for orphan products development are expected to propel boost growth of the global lysosomal storage diseases therapeutics market over the forecast period. Over the last few years, introduction of specific orphan drug regulations has enabled and accelerated research and development of therapies for treatment of several rare diseases. For instance, the USFDA Office of Orphan Products Development (OOPD) focuses on advancing the assessment and development of products (drugs, biologics, devices, or medical foods) that demonstrate potential for diagnosis and/or treatment of rare diseases.

Global Lysosomal Storage Diseases Therapeutics Market: Opportunities 

Use of nanotechnology is expected to offer lucrative growth opportunities for players in the global lysosomal storage diseases therapeutics market. For instance, in October 2019, researchers from University of Santiago de Compostela, Spain, reported development of a cytocompatible formulation based on nanostructured solid lipid carrier systems to deliver the enzyme efficiently to target tissues and successively preserve its activity inside the target cells, chondrocytes, in order to improve treatment of LSDs.

Global Lysosomal Storage Diseases Therapeutics Market: Restraints

Lack of treatment options are expected to limit growth of the market. There exists a significant challenge in transforming scientific discoveries into new treatment options for orphan diseases, due to rarity of these diseases and the challenges associated with conducting clinical trials. Thus, development of new treatment options remains a challenge and also, absence of effective treatments lessens readiness to diagnose and eagerness to study further about the disease.

Key Takeaways:

Enzyme replacement therapy segment in the global lysosomal storage diseases therapeutics market was valued at US$ 6,856.2 Mn in 2019 and is expected to reach US$ 14,938.7 Mn by 2027 at a CAGR of 10.0% during the forecast period. eing an orphan disease condition, regulatory agencies offer the benefit of rapid approval and ease in approval process. This in turn, is expected to expand the overall enzyme replacement therapy market with introduction of new therapies by various key players. For instance, in November 2017, The FDA approved Mepsevii (vestronidase alfa), which is first authorized treatment for pediatric and adult patients suffering from an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII).

The gaucher disease segment held dominant position in the global lysosomal storage diseases therapeutics market in 2018, accounting for 30.0% share in terms of value, followed by Mucopolysaccaridosis and Fabry Diseases, respectively. In July 2017, The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) collaborated to promote the use of innovative approaches in the development of medicines for Gaucher disease. Such strategic collaboration of regulatory bodies for the development of novel innovative drugs is expected to boost the growth of segment over forecast period.

Market Trends

In Latin America, there are considerable variations in requirements and time period for approval of orphan drugs across the countries. For instance, in Brazil, the average time for approval of orphan drug application by ANVISA is 13 months while in Mexico average time taken for orphan drug approval is as low as 5 months.

In Europe, the EU funds over 100 collaborative research projects to address various aspects of rare diseases including LSDs. For instance, the EU offers funding to a European Consortium for Lysosomal Disorders (EUCLYD) – a network of basic and clinical investigators in European countries to study various aspects of LSDs. 


Latin America

Global Lysosomal Storage Diseases Therapeutics Market: Competitive Landscape

Major players operating in the global lysosomal storage diseases therapeutics market include, Shire plc, Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor Pharmaceutical Corp., Protalix Biotherapeutics Inc., Quest Diagnostics, and Amicus Therapeutics, Inc.

Global Lysosomal Storage Diseases Therapeutics Market: Key Developments 

March 2020: Amicus Therapeutics, Inc. started the company’s Global Research and Gene Therapy Center of Excellence in uCity Square in Philadelphia to expand its portfolio of rare disease gene therapy programs

February 2020: Lysogene, a gene therapy platform company, received Fast Track Designation from the U.S. Food and Drug Administration for its LYS-SAF302 program for the treatment of mucopolysaccharidosis Type IIIA, a rare inherited neurodegenerative lysosomal storage disorder


  • By Treatment
    • Enzyme Replacement Therapy
    • Stem Cell Therapy
    • Substrate Reduction Therapy
    • Others
  • By Indication
    • Gaucher's Diseases
    • Fabry Diseases
    • Pompe’s Syndrome
    • Mucopolysaccharidosis
    • Other
  • By End User
    • Hospitals
    • Clinics
  • By Region
    • North America
    • Latin America
    • Europe
    • Asia Pacific
    • Middle East
    • Africa
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