Enzyme Replacement Therapy Market Overview

The enzyme replacement therapy (ERT) market is projected to expand from US$ 9,673.39 million in 2021 to US$ 15,184.70 million by 2028, reflecting a compound annual growth rate (CAGR) of 6.8% from 2022 to 2028.

Market Trends and Growth Drivers

This report outlines the prevailing trends and key factors propelling the growth of the enzyme replacement therapy market. The increase in the global ERT market is primarily driven by the rising incidence of lysosomal storage diseases (LSDs) and the swift regulatory approvals coupled with marketing advantages for drugs designated as orphan drugs. However, intense competition among market participants poses challenges to overall market expansion.

Understanding Enzyme Replacement Therapy

Enzyme replacement therapy is a medical treatment aimed at addressing congenital enzyme deficiencies through the administration of enzymes or proteins that exhibit enzymatic activity. These enzymes can be derived from various sources, including animal, human, and recombinant engineered enzymes. ERT is predominantly utilized for treating rare lysosomal storage disorders such as Pompe's disease, Fabry's disease, Gaucher's disease, Tay-Sachs disease, and Hurler syndrome. The most prevalent method of ERT involves intravenous (IV) infusions, where the replacement enzyme is delivered directly into the bloodstream via a controlled fluid drip.

Regulatory Framework and Orphan Drug Designation

The European Medicines Agency (EMA) plays a crucial role in approving orphan drugs. According to the EMA, once a product with orphan drug designation receives marketing approval for its intended use, it is granted orphan market exclusivity, preventing the approval of similar drugs for the same indication for a period of 10 years.

In March 2016, the EMA introduced the Priority Medicines (PRIME) scheme to expedite the development of product candidates for rare indications with limited or no existing therapies. This scheme offers several advantages to drug developers, including:

• Appointment of a rapporteur from the Committee for Medicinal Products for Human Use (CHMP) to enhance knowledge prior to marketing authorization applications.


• Proactive regulatory dialogue with the EMA to refine development plans and regulatory strategies.


• Provision of scientific advice at critical development milestones, involving stakeholders like health-technology-assessment bodies to facilitate quicker patient access to new treatments.


• Confirmation of the potential for accelerated assessment during marketing authorization applications.

Due to the incentives associated with orphan drug designation, key players in the market are increasingly focusing on developing therapies for orphan diseases. For example, Amicus Therapeutics, Inc. recently received orphan medicinal product designation in Europe from the EMA for Galafold, aimed at treating Fabry disease, and for the combination product ATB200/AT2221 for Pompe disease. Additionally, in September 2020, Amicus Therapeutics was granted PRIME designation for AT-GTX-501, a therapy for CLN6 Batten disease.

Market Segmentation

The global enzyme replacement therapy market is categorized based on enzyme type, therapeutic conditions, route of administration, and end users. In terms of enzyme type, the market includes alglucosidase alfa, agalsidase beta, imiglucerase, idursulfase, galsulfase, velaglucerase alfa, and other enzymes. In 2021, the segment for other enzymes held the largest market share and is anticipated to exhibit the highest CAGR from 2022 to 2028.

When segmented by therapeutic conditions, Gaucher's disease accounted for the largest market share in 2021 and is expected to maintain the highest CAGR during the forecast period. The market is also divided by route of administration into parenteral and oral, with the parenteral segment dominating in 2021 and projected to grow at a higher CAGR. Lastly, by end user, the market is segmented into hospitals, infusion centers, and others, where hospitals held the largest share in 2021, although infusion centers are expected to show the highest growth rate.

Impact of COVID-19 on the Market

The COVID-19 pandemic had a profound impact on the global healthcare landscape. To mitigate the spread of the SARS-CoV-2 virus, governments implemented strict measures that significantly reduced emergency department visits, with a reported decrease of approximately 40% in 2020. Many outpatient appointments and elective procedures were postponed or transitioned to telemedicine.

The disruption of ERT during the pandemic had serious implications for patients with LSDs. Restrictions on hospital visits led to interruptions in scheduled ERTs, exacerbating the conditions of these patients. A survey conducted in 2021 at Sir Ganga Ram Hospital in India revealed that out of 26 patients receiving ERT, 20 experienced significant treatment interruptions, missing an average of eight doses. Transportation issues and delays in medicine importation were cited as major factors contributing to these interruptions, ultimately worsening the health of LSD patients and negatively affecting the enzyme replacement therapy market.

Market Strategies and Developments

Companies in the global enzyme replacement therapy market are employing various organic and inorganic strategies to enhance their market presence. Organic strategies primarily involve product launches and approvals, while inorganic strategies include acquisitions, collaborations, and partnerships. These approaches enable market players to broaden their operations and strengthen their geographic footprint, thereby contributing to overall market growth.

Significant developments in the enzyme replacement therapy market include:

• In May 2022, AbbVie and Cugene Inc. announced an exclusive worldwide license option agreement for CUG252, a potential Treg-selective IL-2 mutein, aimed at treating autoimmune diseases.


• In August 2021, the FDA approved Nexviazyme (avalglucosidase alfa-ngpt) for treating late-onset Pompe disease in patients aged one year and older, demonstrating improvements in respiratory function and walking distance in clinical trials.

Key sources referenced in the preparation of this report include the National Health and Medical Research Council (NHMRC), the Government of Canada, and the Bioprocessing Technology Institute (BTI).