Enzyme Replacement Therapy Market Analysis | 2022-2028

Market Insights

The Enzyme Replacement Therapy Market was estimated at US$ 7.93 billion in 2021 and is expected to grow at a CAGR of 8.28% during 2022-2028 to reach US$ ~13.9 billion in 2028.

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What is enzyme replacement therapy?

Enzyme replacement therapy (ERT) is a medical treatment designed to address enzyme deficiencies in individuals with specific genetic disorders. Typically used for lysosomal storage diseases, ERT involves administering synthetic enzymes intravenously, aiming to replace or supplement the deficient enzymes within the body. These enzymes aid in breaking down accumulated substances within cells, preventing or alleviating symptoms associated with the underlying genetic condition. ERT has shown efficacy in managing diseases such as Gaucher's disease and Fabry disease, improving patients' quality of life by mitigating organ damage and systemic complications associated with enzyme deficiencies.Top of Form

Market Dynamics

The increasing availability of enzyme replacement therapy, increased awareness, and less stringent standards for rare disorders are generating market potential. A rare disease is one that affects fewer than 200,000 people, according to the Genetic and Rare Diseases Information Center. Various organizations are launching strategic initiatives to raise awareness about these diseases and help people live longer lives.

According to Genetic Alliance UK estimates, a single uncommon disease affects over 30,000 people in the UK. Because the majority of chronic diseases are fatal, prompt medical treatment is critical to lowering mortality rates. Gaucher, Fabry, Pompe, various forms of MPS, and SCID disorders are the most common lysosomal storage diseases.

Rare disorders like these occur infrequently. Gaucher's illness, for example, is predicted to affect one in every 100,000 people, according to Orphanet. One of the primary reasons for the pharmaceutical industry's limited growth is the low prevalence of uncommon or orphan diseases.

Several investments/guidelines in the industry have been directed in recent years, which would boost the overall market. Some of them are:

• Rising awareness and an expanding number of market participants venturing into orphan disease management are driving market expansion. Orphan pharmaceuticals used to treat these disorders are expected to thrive during the projected period due to an increase in players investing in R&D to cure these uncommon diseases.

The Orphan Drug Act of 1983 is a public statute in the United States that has been instrumental in the development of innovative medications for the treatment of Tourette syndrome, myoclonus, ALS, muscular dystrophy, and Huntington's disease. Following the act's adoption, numerous regions, including Australia, the European Union, and Japan, followed suit in order to promote improved treatment for orphan illnesses, propelling market expansion.

Segments Analysis

By Enzyme Type

The market is segmented into pancreatic enzymes, agalsidase beta, taliglucerase, velaglucerase alfa, laronidase, alglucosidase alfa, galsulfase, idursulfase, pancreatic enzymes, pegademase, and others. One of the main reasons for the increased frequency of Gaucher disease is the significant market share of enzymes like imiglucerase and velaglucerase alfa.

Pancreatic enzymes such as amylase, lipase, and protease are increasingly being utilized to treat exocrine pancreatic insufficiency (EPI), which is frequent in cancer patients. According to the American Cancer Society, more than 55,440 people are expected to be diagnosed with pancreatic cancer in 2018. Thus, rising pancreatic cancer incidence is likely to boost market growth throughout the predicted period.

Agalsidase beta, which is used to treat Fabry's disease, is expected to grow at a steady CAGR during the projection period. Fabrazyme by Sanofi Genzyme is an enzyme replacement medicine used to treat hereditary diseases. It works by replacing the enzyme responsible for low levels of GL-3, which is missing or defective in Fabry's disease patients. Because the majority of these diseases are inherited, early treatment is critical to lowering mortality rates. Rising disease awareness has contributed to an increase in demand for enzyme replacement treatment solutions. As a result, enzymes are expected to grow rapidly during the forecast period.

By Disease Indication Type

The market is divided into Gaucher disease and others. Fabry disease, Pompe disease, SCID, MPS, and others. The MPS segment is further divided into MPS I - Hurler, Hurler Scheie and Scheie, MPS II – Hunter, and MPS III – Sanfilippo. The high prevalence of Gaucher's disease is due to its considerable market share. Gaucher disease is an inherited condition caused by a recessive gene mutation. According to the National Library of Medicine, the disease is predicted to affect one in every 50,000 to 100,000 people in the general population, depending on the region. According to Clinicaltrials.gov, around 60 studies for Gaucher's disease are now underway, with pharmaceutical companies funding the majority of them. Diseases such as Fabry, Pompe, and MPS are also gaining traction in the market as a result of increased awareness generated by organizations such as NORD (National Organization for Rare Disorders), Global Genes, GARD (Genetic and Rare Diseases Information Center), and the National Institutes of Health, among others.

Regional Analysis

The enzyme replacement therapy market is broken down geographically into areas like North America, Europe, Asia-Pacific, and the Rest of the World (RoW). In 2021, North America led the market. The presence of prominent market participants in North America and well-developed healthcare infrastructure have propelled the regional segment's rise. Furthermore, the region's embrace of sophisticated technology and increased focus on medication research are projected to promote industry growth.

After North America, the European region accounted for the second-highest revenue. The European region's healthcare system is publicly funded, and some countries have implemented universal healthcare systems. The existence of a large number of MPS and SCID patients is one of the reasons driving market expansion in the European region.

According to Shire Outcome Surveys' Fabry Outcome Survey data, Germany, Japan, and the United Kingdom have the highest prevalence of Fabry's disease. According to data from the FDA's Novel Drug Approvals, more than 39% of novel medications authorized in 2017 were for orphan diseases. These and other factors are expected to drive regional market expansion even further. The Asia Pacific region includes both industrialized and emerging countries such as China, India, and others. The majority of the countries in this region are economically developing, with rising disposable income. Many global corporations are making significant investments in this region. Furthermore, countries such as Japan are technologically sophisticated in healthcare and medical research, which helps the industry.

Key Players

The market has a large number of both global and local companies. The bulk of these market participants has a well-established portfolio of lysosomal storage diseases, making treatment simple and efficient. Key players are primarily concerned with discovering and marketing medications for rare and hereditary disorders.

For example

• Shire, plc has over 40 medications distributed globally that are completely focused on rare and orphan diseases. The company is also committed to raising awareness about these disorders and, as a result, is continually launching new campaigns to increase awareness. Rare Count is one such imitation that the business debuted in February 2017. This project was developed in order to raise public awareness of orphan disease.

The overall competitive landscape has been affected due to these mergers and acquisitions. The following are the major players in the enzyme replacement therapy market:

• AbbVie
• Actelion (Janssen)
• Alexion Pharmaceuticals Inc.
• Allergan plc
• Amicus Therapeutics, Inc.
• Biomarin Pharmaceutical Inc.
• Horizon Pharma Public Limited Company
• Protalix Biotherapeutics
• Recordati Rare Diseases
• Shire Plc
• Sanofi S.A

Research Methodology

This strategic assessment report, from Stratview Research, provides a comprehensive analysis that reflects today’s enzyme replacement therapy market realities and future market possibilities for the forecast period. The report segments and analyzes the market in the most detailed manner in order to provide a panoramic view of the market. The vital data/information provided in the report can play a crucial role for the market participants as well as investors in the identification of the low-hanging fruits available in the market as well as to formulate the growth strategies to expedite their growth process.

This report offers high-quality insights and is the outcome of a detailed research methodology comprising extensive secondary research, rigorous primary interviews with industry stakeholders, and validation and triangulation with Stratview Research’s internal database and statistical tools. More than 1,000 authenticated secondary sources, such as company annual reports, fact books, press releases, journals, investor presentations, white papers, patents, and articles, have been leveraged to gather the data. We conducted more than 15 detailed primary interviews with the market players across the value chain in all four regions and industry experts to obtain both qualitative and quantitative insights.

Report Features

This report provides market intelligence in the most comprehensive way. The report structure has been kept such that it offers maximum business value. It provides critical insights into the market dynamics and will enable strategic decision-making for the existing market players as well as those willing to enter the market.

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Customization Options

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• Detailed profiling of additional market players (up to three players)
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• Benchmarking of key players on the following parameters: Product portfolio, geographical reach, regional presence, and strategic alliances

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