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Global Lysosomal Disease Treatment Market Report 2022 to 2028: Featuring Pfizer, Takeda Pharmaceutical, Sanofi and Novartis Among Others -

PRESS RELEASE: Paid content from Business Wire
Press release content from Business Wire. The AP news staff was not involved in its creation.
January 10, 2023 GMT

DUBLIN--(BUSINESS WIRE)--Jan 10, 2023--

The “Global Lysosomal Disease Treatment Market Size, Share & Industry Trends Analysis Report By Disease Type (Gaucher’s Diseases, Fabry Diseases, Mucopolysaccharidosis, Pompe’s Syndrome), By Type of Therapy By End User, By Regional Outlook and Forecast, 2022 - 2028” report has been added to’s offering.

The Global Lysosomal Disease Treatment Market size is expected to reach $11 billion by 2028, rising at a market growth of 6.3% CAGR during the forecast period.

Lysosomal diseases (LSDs) are a complex group of metabolic disorders that are often inherited in an autosomal recessive manner. There are currently 45 known diseases. They are caused by malfunctions in various lysosomal processes, most often by abnormalities in genes that produce catabolic enzymes responsible for the breakdown of macromolecules.

As the faulty enzyme accumulates substrate, cell malfunction and clinical symptoms escalate. Other types of lysosomal dysfunction that can lead to lysosomal storage include defects in post-translational processing of lysosomal enzymes, errors in enzyme trafficking or targeting, and dysfunction of non-enzymatic transmembrane and solubilize.


Lysosomal hydrolase mutations, which are implicated in the breakdown of cellular macromolecules, are the primary cause of LSDs, accounting for more than two-thirds of cases. As a result, there are many different types of lipids, glycogen, mucopolysaccharides, and glycoproteins among the storage products in LSDs that are biochemical in origin. The LSDs are all inherited as autosomal recessive characteristics, except the X-linked Fabry, Danon, and Hunter diseases.

The LSDs can affect the majority of organs, either alone or as part of a multisystem illness, and are clinically very heterogeneous. Even though lysosomal enzymes are housekeeping genes and expressed in the majority of cells, the tissues on which they act are distributed much less uniformly, and this determines which tissues in any individual are affected.

Market Growth Factors

Incidence of Lysosomal Disorders Are Rising

The primary driving forces in the enzyme replacement therapy market are the expanding research and development for lysosomal disease diagnosis and drug development for treatment. The increasing incidence of lysosomal disease will be a key factor contributing to the acceleration of the market’s growth rate.


According to a report published by the National Center for Biotechnology Information, the prevalence of Fabry disease in communities of white, male individuals ranged from 1:17,000 to 1:117,000 in January 2020. Atypical presentations were associated with around 1:6000-1:40,000 females and 1:1000-1:3000 males, whereas 1:22,000-1:40,000 males had the classic Fabry disease mutations.

Financial Incentives for Developing Orphan Drugs to Recoup R&D Expenses

The increases in healthcare spending, which helps to improve its infrastructure, is a crucial element affecting the market’s growth rate for therapies for lysosomal diseases. The market dynamics will be further impacted by various government organizations’ efforts to strengthen the healthcare infrastructure by boosting funding. Additionally, the market for lysosomal disease therapies will grow as a result of the rising acceptance rate of early diagnosis and the increasing number of government programs to raise awareness. Along with this, expanding government benevolent programs and an increase in disposable money is expected to accelerate market expansion.


Marketing Restraining Factor:

The Treatment of Stem Cells Is Fraught with Issues

Many of the issues that can arise soon after the transplant are caused by the bone marrow being destroyed just before it by drugs or radiation. Some may be unintended consequences of the conditioning procedures themselves. The patient can get support from the transplant team to manage side effects. Most can be addressed to make the patient feel better, and some can be prevented. This is not a comprehensive list, so please inform the physician or the transplant team of any issues or adjustments that experience.


Scope of the Study

Market Segments Covered in the Report:

By Disease Type

  • Gaucher’s Diseases
  • Fabry Diseases
  • Mucopolysaccharidosis
  • Pompe’s Syndrome
  • Others

By Type of Therapy

  • Enzyme Replacement Therapy
  • Substrate Reduction Therapy
  • Stem Cell Therapy
  • Others

By End User

  • Hospitals
  • Clinics
  • Others

By Geography

  • North America
    • US
    • Canada
    • Mexico
    • Rest of North America
  • Europe
    • Germany
    • UK
    • France
    • Russia
    • Spain
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • South Korea
    • Singapore
    • Malaysia
    • Rest of Asia Pacific
    • Brazil
    • Argentina
    • UAE
    • Saudi Arabia
    • South Africa
    • Nigeria
    • Rest of LAMEA

Key Market Players

List of Companies Profiled in the Report:

  • Merck & Co., Inc.
  • Johnson & Johnson (Janssen Global Services, LLC)
  • Eli Lilly And Company
  • Pfizer, Inc.
  • Takeda Pharmaceutical Company Limited
  • Sanofi S.A.
  • Novartis AG
  • AstraZeneca PLC (Alexion Pharmaceuticals, Inc.)
  • BioMarin Pharmaceutical Inc.
  • Sigilon Therapeutics, Inc.

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SOURCE: Research and Markets

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PUB: 01/10/2023 09:42 AM/DISC: 01/10/2023 09:41 AM