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 tohave a frequency of one in every 50,000 live births. Most LSDs are caused dueto autosomal recessive inheritance leading to genetic mutations resulting in deficiencyof lysosomal enzymes.
The global LysosomalStorage 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 Mnby the end of 2027.
Global Lysosomal Storage DiseasesTherapeutics Market: Drivers
Favorable regulations for orphanproducts development are expected to propel boost growth of the globallysosomal storage diseases therapeutics market over the forecast period. Overthe last few years, introduction of specific orphan drug regulations hasenabled and accelerated research and development of therapies for treatment ofseveral rare diseases. For instance, the USFDA Office of Orphan ProductsDevelopment (OOPD) focuses on advancing the assessment and development ofproducts (drugs, biologics, devices, or medical foods) that demonstratepotential for diagnosis and/or treatment of rare diseases.
Global Lysosomal Storage DiseasesTherapeutics Market: Opportunities
Use of nanotechnology is expectedto offer lucrative growth opportunities for players in the global lysosomalstorage diseases therapeutics market. For instance, in October 2019,researchers from University of Santiago de Compostela, Spain, reporteddevelopment of a cytocompatible formulation based on nanostructured solid lipidcarrier systems to deliver the enzyme efficiently to target tissues andsuccessively preserve its activity inside the target cells, chondrocytes, inorder to improve treatment of LSDs.
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Global Lysosomal Storage DiseasesTherapeutics Market: Restraints
Lack of treatment options areexpected to limit growth of the market. There exists a significant challenge intransforming scientific discoveries into new treatment options for orphandiseases, due to rarity of these diseases and the challenges associated withconducting clinical trials. Thus, development of new treatment options remainsa challenge and also, absence of effective treatments lessens readiness todiagnose and eagerness to study further about the disease.
Key Takeaways:
Enzyme replacement therapysegment in the global lysosomal storage diseases therapeutics market was valuedat US$ 6,856.2 Mn in 2019 and is expected to reach US$ 14,938.7 Mn by 2027 at aCAGR of 10.0% during the forecast period. eing an orphan disease condition,regulatory agencies offer the benefit of rapid approval and ease in approvalprocess. This in turn, is expected to expand the overall enzyme replacementtherapy market with introduction of new therapies by various key players. Forinstance, in November 2017, The FDA approved Mepsevii (vestronidase alfa),which is first authorized treatment for pediatric and adult patients sufferingfrom an inherited metabolic condition called mucopolysaccharidosis type VII(MPS VII).
The gaucher disease segment helddominant position in the global lysosomal storage diseases therapeutics marketin 2018, accounting for 30.0% share in terms of value, followed byMucopolysaccaridosis and Fabry Diseases, respectively. In July 2017, TheEuropean Medicines Agency (EMA) and the United States Food and DrugAdministration (FDA) collaborated to promote the use of innovative approachesin the development of medicines for Gaucher disease. Such strategiccollaboration of regulatory bodies for the development of novel innovative drugsis expected to boost the growth of segment over forecast period.
Market Trends
In Latin America, there areconsiderable variations in requirements and time period for approval of orphandrugs across the countries. For instance, in Brazil, the average time forapproval of orphan drug application by ANVISA is 13 months while in Mexicoaverage time taken for orphan drug approval is as low as 5 months.
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In Europe, the EU funds over 100collaborative research projects to address various aspects of rare diseasesincluding LSDs. For instance, the EU offers funding to a European Consortiumfor Lysosomal Disorders (EUCLYD) – a network of basic and clinicalinvestigators in European countries to study various aspects of LSDs.
Regulations
Latin America
Global Lysosomal Storage DiseasesTherapeutics Market: Competitive Landscape
Major players operating in theglobal lysosomal storage diseases therapeutics market include, Shire plc,Pfizer, Inc., Sanofi, BioMarin Pharmaceutical Inc., Actelion Ltd., Raptor PharmaceuticalCorp., Protalix Biotherapeutics Inc., Quest Diagnostics, and AmicusTherapeutics, Inc.
Global Lysosomal Storage DiseasesTherapeutics Market: Key Developments
March 2020: Amicus Therapeutics,Inc. started the company’s Global Research and Gene Therapy Center ofExcellence in uCity Square in Philadelphia to expand its portfolio of raredisease gene therapy programs
February 2020: Lysogene, a genetherapy platform company, received Fast Track Designation from the U.S. Foodand Drug Administration for its LYS-SAF302 program for the treatment ofmucopolysaccharidosis Type IIIA, a rare inherited neurodegenerative lysosomalstorage disorder
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