Enzyme Replacement Therapy Market Size, Share & Trends Analysis Report By Product (Agalsidase Beta, Imiglucerase, Velaglucerase Alfa, Idursulfase, Galsulfase, Laronidase, Others), By Disease (Gaucher Disease, Fabry Disease, Pompe Disease, Mucopolysaccharidosis, Exocrine Pancreatic Insufficiency (EPI), Others), By End-User (Hospitals, Infusion Centers and Home Healthcare Setting), By Route of Administration (Oral, Parenteral) and By Region(North America, Europe, APAC, Middle East and Africa, LATAM) Forecasts, 2024-2032

Enzyme Replacement Therapy Market Size

The global enzyme replacement therapy market size was valued at USD 9.25 billion in 2023 and is projected to reach USD 18.42 billion by 2032, growing at a CAGR of 8.98% during the forecast period (2024-2032).

Enzyme replacement therapy is a type of medical care in which a missing or deficient enzyme in the body is replaced. This procedure is typically carried out by administering an intravenous (IV) infusion of an enzyme-containing solution to the patient. Enzyme Replacement Therapy (ERT) is a medical procedure that treats people with specific genetic illnesses by substituting any lacking or insufficient enzymes. The primary conditions ERT treats are lysosomal storage disorders (LSDs), a group of rare hereditary metabolic diseases characterized by chemical buildup inside cells because of enzyme deficiencies. Recombinant enzymes are administered intravenously as part of ERT to replace the missing or insufficient enzyme. The enzyme specifically targets and breaks down the chemicals that are building up, reducing their concentrations and accelerating the onset of symptoms and disease.

Enzyme Replacement Therapy Market Driving Factors

Shift of patients to homecare settings due to cost-effectiveness and better quality of life

Patients with lysosomal storage diseases more frequently receive care at home than in hospitals. For instance, an Environmental Research and Public Health study revealed that 80% of the patients polled in Poland preferred receiving care from a qualified nurse at home. Most patients are reportedly willing to switch from hospital care to home settings for treatment because of the latter's safety, efficacy, and higher quality of life. Additionally, the rise of COVID-19 has contributed significantly to the growing trend of home care settings. A Galenos Publishing House survey analysis found that 89% of respondents sought home care therapy during the pandemic because they feared contracting the disease in hospital settings. Homecare treatment's particular benefits, including its comfort, cost-effectiveness, and risk-free environment, drive the pandemic's migration of patients toward these settings. Such factors drive market growth.

Increasing prevalence of rare diseases

The prevalence of several lysosomal storage disorders, such as Gaucher, Fabry, Pompe, and MPS, is gradually rising globally. For instance, the National Institute of Neurological Disorders and Stroke reported in August 2021 that there were approximately 32,950 cases of Pompe disease in the United States or 1 in 40,000 people. The National Fabry Foundation also estimates there were around 7,713 Fabry patients in the United States as of May 2020. Therefore, there is a greater demand for effective treatment alternatives, such as enzyme replacement therapy, due to a large patient pool with uncommon lysosomal storage disorders. A progressive increase in the diagnosis rate of uncommon diseases is also a result of growing efforts by governments and non-profit groups in various nations to raise public awareness. As a result, these crucial elements, as well as favorable reimbursement policies and government support for medical care in developed nations, are increasing the rate of this therapy's adoption and propelling the market's expansion over the forecast period.

Market Restraining Factors

High cost of treatment

Positive reimbursement policies available in industrialized nations and the increased occurrence of uncommon illnesses are key drivers of market expansion. However, the market expansion is constrained by several problems, including a shortage of qualified healthcare technicians for this therapy and inadequate reimbursement policies in developing nations. For instance, the National Gaucher Foundation estimates that the average annual expenditure for one patient with Gaucher disease is about USD 0.2 million. A considerable delay was seen between the commencement and initiation of the ERT process symptoms in the afflicted population in Brazil, according to a report by Elsevier B.V. The absence of insurance coverage in this nation mostly brought on this delay.

Also, there is a stronger demand for alternative therapies than ERT procedures due to the availability of these therapies, such as chaperone therapy, in which pharmacological chaperones can effectively pass the human body's blood-brain barrier. The aforementioned problems and several adverse effects, such as pyrexia, flushing, dyspnea, and others within the first 1-4 months of treatment, further restrain the global market growth for enzyme replacement therapy.

Market Opportunities

Wide product offerings by key players

A select group of well-known companies like BioMarin, Sanofi, AbbVie Inc., and Takeda Pharmaceutical Company Limited dominate the industry. One of the main factors influencing these companies' dominance in the industry is their great emphasis on partnerships and acquisitions with other large market players to broaden the product's reach internationally. For instance, Takeda Pharmaceutical Company Limited stated in September 2021 that it had entered into a partnership and collaboration agreement with JCR Pharmaceuticals Co., Ltd. to commercialize JR-141 outside of the United States to treat Hunter syndrome.

On the other hand, up-and-coming firms like Clinigen Group plc, TEIJIN LIMITED, and others are continually concentrating on new product introductions for various therapeutic diseases. Additionally, they are helping to meet the growing demand for the treatment of lysosomal storage disorders in both developed and emerging nations by placing a major emphasis on getting marketing approval from regulatory authorities. For instance, in May 2019, TEIJIN LIMITED introduced Revcovi 2.4 mg, intending to treat Adenosine Deaminase (ADA) Deficiency via Parenteral Dosage.

Regional Insights

North america dominates the global market

North America is the most significant global enzyme replacement therapy market shareholder and is expected to grow substantially over the forecast period. North America is expected to hold a major market share of 40.84% due to actions taken by the government and other organizations on the treatment of rare diseases. For instance, the National Institutes of Health funded over USD 31 million to 20 teams in 2019—including five new groups of researchers, doctors, patients, and families—to study rare diseases. The data coordination centers have given an additional USD 7 million to support the research initiatives. Additionally, one of the National Institute of Health's institutions, the National Center for Advancing Translational Sciences (NCATS), is completely focused on treating people with rare diseases. The use of enzyme replacement treatment in North America is also anticipated to increase due to the greater prevalence of uncommon disorders. Approximately 25 to 35 million Americans currently suffer from rare diseases, according to the Genetic and Rare Diseases Information Center (GARD).

Europe: fastest growing region with the highest cagr

Europe held the second-largest market share worldwide. Favorable reimbursement practices in a few European nations for several uncommon lysosomal illnesses, including Gaucher disease, MPS, and others, are principally responsible for the rise. For instance, a patient received the first reimbursement in ERT for Fabry disease under the Polish National Health Fund's national drug program in 2019, according to F1000 Research Ltd. The market in this region is expanding due to the rapid development of the healthcare infrastructure and the rising demand for treatments for uncommon diseases. During the projection period, Asia Pacific is anticipated to develop at a considerable CAGR. Growing patient populations with uncommon disorders and increased government and other private organization efforts to raise knowledge of this therapy are boosting demand and adoption among the general public and propelling the market's expansion.

Enzyme Replacement Therapy Market Segmentation Analysis

By product

The market is bifurcated into Agalsidase Beta, Imiglucerase, Velaglucerase Alfa, Idursulfase, Galsulfase, Laronidase, and others. The Agalsidase beta segment dominates the global market and is expected to grow at a CAGR of 8.23% over the forecast period. This is due to the rising prevalence of chronic and fatal lysosomal storage diseases. Lysosomal storage disorders, such as Fabry, are extremely rare X-linked diseases. Fabry disease is being treated with Algasidase Alfa, a recombinant version of human a-galactosidase (AGAL). This enzyme is produced under Replagal by the Shire, a Takeda Pharmaceutical Company affiliate. Replagal has been given the go-ahead for marketing and manufacturing in Canada, the UK, and other European nations.

In addition, the study published in JIMD in 2019 found that Fabry disease is more common in people receiving dialysis. The Fabry disease has been identified in 1.2% of dialysis patients worldwide. Due to the rising frequency of Fabry disease, treatment is now necessary, which is anticipated to drive growth in the market for enzyme replacement therapy in the algasidase alfa segment.

By disease type

The market is segmented into Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidosis, exocrine pancreatic insufficiency (EPI), and others. The enzyme replacement therapy market treats various diseases, but lysosomal storage diseases, including Gaucher disease, Fabry disease and Pompe disease, reign supreme. Gaucher disease currently leads the pack due to its slightly higher prevalence and need for lifelong treatment, factors that drive market share. All lysosomal storage disorders, however, are dominating due to their high costs and ongoing treatment requirements. Lysosomal storage disorders are predicted to continue to be important actors for the foreseeable future, despite the possibility that developments in the treatment of other diseases might alter the situation.

By route of administration

The market is segmented into oral and parenteral. Parenteral administration, particularly intravenous (IV) infusions, is the predominant mode of delivery in the field of enzyme replacement therapy. Many significant benefits are the source of this domination. Firstly, compared to oral medications, parenteral administration allows for the direct delivery of enzymes into the bloodstream, resulting in a more rapid and regulated release.  This is critical for many enzyme replacement therapies, especially those aimed at rapid relief of symptoms or prevention of disease progression. Second, oral medications can break down or be poorly absorbed by the digestive system, making them less effective. Parenteral administration bypasses these problems, ensuring that target organs reach a more reliable and predictable dose.

By end user

The market is segmented into hospitals, infusion centers, and others. Enzyme replacement treatment is mostly provided by hospitals. There are several reasons for this dominance. Initially, these therapies are frequently intricate processes requiring specialized tools and skilled workers—resources that are easily accessible in medical facilities. Enzyme replacement, in contrast, frequently necessitates intravenous (IV) treatment in a sterile, controlled setting. Second, patient monitoring is very important because of potential side effects, and hospitals have both the staff and resources to ensure patient safety. Finally, the severity of treatable conditions such as Gaucher disease often require extensive medical care provided by hospitals. Although infusion centers offer potential convenience in the future and home care may be appropriate for some stable patients, hospitals will remain the primary provider of enzyme replacement therapy for the foreseeable future.

Recent Developments

• April 2023- According to a statement from AbbVie, QULIPTA® (atogepant) was given FDA approval to expand its indication to include the prevention of migraine in adults. As a result of the approval, QULIPTA is now the only oral calcitonin gene-related peptide (CGRP) receptor antagonist authorized to treat both episodic and persistent migraine. Chronic migraine sufferers report 15 or more headache days per month, with at least eight of those days being migraine days.
• March 2023- The U.S. Food and Drug Administration (FDA) accepted a supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection, extending treatment in the country to include children with achondroplasia under the age of five. This news was made public by BioMarin Pharmaceutical Inc., a global biotechnology company committed to transforming lives through genetic discovery. The most frequent cause of disproportionately small stature is achondroplasia.