Enzyme Replacement Therapy Market Size, Share, Opportunities, And Trends By Drug Class (Agalsidase, Laronidase, Elosulfasealfa, Galsulfase, Others), By Indication (Type 1 Gaucher Disease, Prompe Disease, MPS, Exocrine Pancreatic Insufficiency, Fabry Disease, Others), By End-User (Hospitals & Clinics, Homecare Settings & Infusion Centers), And By Geography - Forecasts From 2023 To 2028

  • Published : Aug 2023
  • Report Code : KSI061616032
  • Pages : 145

During the forecast period, the enzyme replacement therapy market is anticipated to grow. A person with a hereditary enzyme deficiency syndrome receives enzyme replacement therapy (ERT) to replace a missing or defective enzyme. Through intravenous (IV) or oral administration, it restores the patient's body's depleted enzyme levels. The IV method, which delivers the replacement enzyme straight into the circulation through a carefully regulated fluid flow, is well-liked by patients.

Factors that will boost the demand for enzyme replacement therapy

Growth in the incidence of chronic, fatal lysosomal storage disorders including Gaucher, Pompe, Fabry, and Scid is a key factor driving the demand for enzyme replacement therapy worldwide. Additionally, the government's measures to raise public knowledge of the availability of ERT are anticipated to make a significant contribution to the expansion of the worldwide enzyme replacement therapy industry. The enzyme replacement therapy market for enzyme replacement treatment is anticipated to grow profitably as a result of new therapeutic breakthroughs and increased product pipeline analyses.

High prevalence of rare diseases

Lysosomal storage diseases including Gaucher, Fabry, Pompe, and MPS are becoming more prevalent on a global scale. According to statistics released by the National Institute of Neurological Disorders and Stroke in August 2021, there are an estimated 32,950 instances of Pompe disease in the United States, affecting one in 40,000 persons. The National Fabry Foundation estimates that there were around 7,713 Fabry patients in the United States as of May 2020. There is a greater need for efficient treatment options, such as enzyme replacement therapy due to the high prevalence of rare lysosomal storage diseases which is predicted to upsurge the enzyme replacement therapy market.

Technological advancements in the enzyme replacement therapy market

Technological developments have accelerated the enzyme replacement therapy market growth. Significant advancements in biotechnology have led to the creation of more complex and specialist enzyme replacement therapies. Recombinant enzymes are now being manufactured with superior properties such as increased stability, and specificity owing to developments in recombinant DNA technology. These scientific advancements have improved the efficacy and safety profiles of ERT medications, improving their capacity to address underlying enzyme abnormalities and improving patient outcomes. Future research into gene therapy and other cutting-edge treatment modalities may increase the effectiveness of ERT.

The shift of patients to homecare settings

Lysosomal storage disease patients are treated at home more commonly than in hospitals. For instance, according to research by the Environmental Research and Public Health Department 80% of the patients polled in Poland preferred receiving treatment from a licensed nurse at home. According to reports, the majority of patients are open to switching from hospital care to home settings for treatment because the latter is safer, more effective, and provides a greater quality of life. Patients are migrating towards homecare therapy because of its unique advantages, including its comfort, cost-effectiveness, and risk-free atmosphere throughout the pandemic. These elements fuel enzyme replacement therapy industry expansion.

Increased government financing

The detection rate of unusual diseases gradually rises as a result of growing government and nonprofit activities in several nations to promote public awareness regarding rare diseases. The rate of adoption of enzyme replacement therapy is therefore rising due to important variables including favorable reimbursement policies and government funding for treatment in industrialized countries. However, a lack of skilled medical professionals for this therapy and inadequate reimbursement laws in developing countries are restricting the enzyme replacement therapy market growth.

The enzyme replacement market is anticipated to propel in the North American region.

North America’s highly developed healthcare industry and the presence of major enzyme replacement therapy market players are the key factors anticipated to propel the enzyme replacement therapy market. Additionally, the region's growing focus on medication research and adoption of cutting-edge technology is anticipated to fuel enzyme replacement therapy market growth. The second-highest revenue-producing area after North America was Europe, which is anticipated to expand. The healthcare system in Europe is backed by the government, and some of the European countries have universal healthcare programs in place. The presence of numerous patients with MPS and SCID is one of the key drivers of enzyme replacement therapy market growth in the European area.

Product innovation by key players

There are several local and international companies in the enzyme replacement therapy market Shire Plc, AbbVie, and Allergan plc are a few of the top market participants that have a significant presence in terms of a distribution network and operational facilities. The primary focus of the major players is on creating and promoting medicines that are intended to treat rare and hereditary disorders. For instance, Shire, plc sells approximately 40 medications that are completely focused on treating rare and orphan illnesses and are available worldwide. The firm continually runs different initiatives to promote awareness since it is likewise committed to raising considerable awareness about these disorders.

Government Regulations

  • Enzyme replacement treatments are subject to rules set out by the U.S. Food and Drug Administration (FDA), which demand that producers submit safety and effectiveness information for each product before approving it for use in patients.
  • Information on all European regulatory standards about enzyme replacement treatment can be found in one location due to the European Regulatory Network (ERN). It is in charge of enabling the mutual recognition of products and harmonizing rules across member nations.,
  • Enzyme replacement treatments are subject to restrictions set down by the European Medicines Agency (EMA), which demand that manufacturers provide thorough documentation of the effectiveness and safety of their medicines before they may be authorized for use in patients.

Market Key Developments

  • In February 2023, the FDA authorized Lamzede (velmanase alfa), the first enzyme replacement medication licensed in the U.S. for the treatment of the non-central nervous system aspects of the condition.
  • In June 2022, Sanofi's enzyme replacement medicines, "Nexviadyme" for Pompe disease and "Xenpoxyme" for treating ASMD (acid sphingomyelinase deficiency) non-central nervous system symptoms, received approval from the European Commission.
  • In May 2022, an exclusive worldwide license option agreement for CUG252, a potential best-in-class Treg-selective IL-2 mutein, as well as other novel IL-2 muteins, has been announced by AbbVie and Cugene Inc., a clinical-stage biotechnology company focused on developing next-generation precision immunology and oncology medicines to treat autoimmune disease and cancer.


  • By Drug Class
    • Agalsidase
    • Laronidase
    • Elosulfasealfa
    • Galsulfase
    • Others
  • By Indication
    • Type 1 Gaucher Disease
    • Prompe Disease
    • MPS
    • Exocrine Pancreatic Insufficiency
    • Fabry Disease
    • Others
  • By End-User
    • Hospitals & Clinics
    • Homecare Settings & Infusion Centers
  • By Geography
    • North America
      • United States
      • Canada
      • Mexico
    • South America
      • Brazil
      • Argentina
      • Others
    • Europe
      • United Kingdom
      • Germany
      • France
      • Spain
      • Others
    • Middle East and Africa
      • Saudi Arabia
      • UAE
      • Israel
      • Others
    • Asia Pacific
      • Japan
      • China
      • India
      • South Korea
      • Indonesia
      • Thailand
      • Others


1.1. Market Overview

1.2. Market Definition

1.3. Scope of the Study

1.4. Market Segmentation

1.5. Currency

1.6. Assumptions

1.7. Base, and Forecast Years Timeline


2.1. Research Data

2.2. Assumptions


3.1. Research Highlights


4.1. Market Drivers

4.2. Market Restraints

4.3. Porter’s Five Force Analysis

4.3.1. Bargaining Power of Suppliers

4.3.2. Bargaining Power of Buyers

4.3.3. Threat of New Entrants

4.3.4. Threat of Substitutes

4.3.5. Competitive Rivalry in the Industry

4.4. Industry Value Chain Analysis


5.1. Introduction

5.2. Agalsidase

5.3. Laronidase

5.4. Elosulfasealfa

5.5. Galsulfase

5.6. Others


6.1. Introduction

6.2. Type 1 Gaucher Disease

6.3. Prompe Disease

6.4. MPS

6.5. Exocrine Pancreatic Insufficiency

6.6. Fabry Disease

6.7. Others


7.1. Introduction

7.2. Hospitals & Clinics

7.3. Homecare Settings & Infusion Centers


8.1. Introduction

8.2. North America

8.2.1. United States

8.2.2. Canada

8.2.3. Mexico

8.3. South America

8.3.1. Brazil

8.3.2. Argentina

8.3.3. Others

8.4. Europe

8.4.1. United Kingdom

8.4.2. Germany

8.4.3. France

8.4.4. Spain

8.4.5. Others

8.5. The Middle East and Africa

8.5.1. Saudi Arabia

8.5.2. UAE

8.5.3. Israel

8.5.4. Others

8.6. Asia Pacific

8.6.1. Japan

8.6.2. China

8.6.3. India

8.6.4. South Korea

8.6.5. Indonesia

8.6.6. Thailand

8.6.7. Others


9.1. Major Players and Strategy Analysis

9.2. Market Share Analysis

9.3. Mergers, Acquisitions, Agreements, and Collaborations

9.4. Vendor Competitiveness Matrix


10.1. Fierce Pharma

10.2. Cerezyme

10.3. Sanofi

10.4. Mayo Clinic

10.5. BioMarin Pharmaceuticals

10.6. Antozyme Biotech

10.7. Optum

10.8. Takeda Pharmaceuticals

10.9. Creon

Fierce Pharma



Mayo Clinic

BioMarin Pharmaceuticals

Antozyme Biotech


Takeda Pharmaceuticals