Enzyme Replacement Therapy Market Size, Share & Industry Analysis, By Drug Class (Alglucosidase alfa, Agalsidase, Pancrelipase, Idursulfase, Laronidase, Imiglucerase, Elosulfase alfa, Asfotasealfa, Galsulfase, Velaglucerase alfa, and Others), By Route of Administration (Parenteral and Oral), By Indication (Type 1 Gaucher Disease, Pompe Disease, MPS (Mucopolysaccharidosis), Exocrine Pancreatic Insufficiency (EPI), Fabry Disease, Hypophosphatasia, and Others), By End User (Hospitals and Homecare Settings & Infusion Centers), and Regional Forecast, 2024-2032

The global enzyme replacement therapy market size was USD 13.45 billion in 2023. The market is projected to grow from USD 14.61 billion in 2024 to USD 29.49 billion by 2032 at a CAGR of 9.2% during the 2024-2032 period. North America dominated the enzyme replacement therapy market with a market share of 47.96% in 2023.

Enzyme replacement therapy is a process in which different types of enzymes are administered in patients to overcome enzyme deficiencies or related malfunction. The rising prevalence of rare, chronic, and inherited disorders, including various kinds of lysosomal storage diseases and genetic disorders is fostering the demand for effective treatment options. According to data published by Cleveland Clinic, it was estimated that around 6,000 people in the U.S. were suffering from Gaucher disease and around 95% of them had type 1 Gaucher in 2020.

Currently, major market players such as BioMarin, Sanofi, and Takeda Pharmaceutical Company Limited. are constantly focusing on introducing advanced therapy options with different drug classes in the market to cater to the increasing demand for this therapy. For instance, in July 2019, Takeda Pharmaceutical Company Limited introduced Idursulfase, Velaglucerase Alpha, and Agalsidase Alfa for the treatment of hunter syndrome, Gaucher disease and Fabry disease in India to meet the demand for therapy options among patients suffering from rare diseases.

Thus, a significant rise in the prevalence of rare disorders and the increasing number of products being approved in the market are driving the product adoption among the patient population. This, other factors such as improving healthcare infrastructure and favorable reimbursement policies for this therapy are increasing the product demand and adoption.

During the COVID-19 pandemic, the global market was positively impacted due to the huge growth in the sales of drugs used in this therapy by key players. Furthermore, increased initiatives by healthcare professionals to provide training sessions to patients for self-administration prevented delays in treatment during the COVID-19 pandemic. Additionally, from 2022 onwards, the market has consistently grown due to the increasing sales of drugs. It will continue to grow during the forecast period with the rising demand for these types of therapies.

Enzyme Replacement Therapy Market Trends

Shift of Patients to Homecare Settings Due to Cost-effectiveness and Better Quality of Life

Recently, a shift from hospital to home care settings is being observed among patients diagnosed with lysosomal storage disorders.

For instance, according to a research article by Environmental Research and Public Health, it was reported that 80% of the surveyed patients in Poland preferred home-based treatment by a trained nurse. Also, it was stated that due to the safety, efficacy, and better quality of life in the home settings, the majority of the patients are willing to shift from hospital care to home settings for their treatment.

Also, the emergence of COVID-19 has been instrumental in the increasing shift toward home care settings. According to a survey analysis published by Galenos Publishing House, 89% of participants preferred to receive home care treatment during the pandemic due to the fear of getting infected in hospital settings. Thus, distinct advantages offered by homecare treatment such as comfort, cost-effective in nature, and risk-free environment are directing the shifts of patients toward homecare settings in the pandemic.

Furthermore, the increasing interest of patients to undergo enzyme replacement therapy at home with diseases, such as Lysosomal Storage Diseases (LSDs) and metabolic disorders is propelling the growth of the market.

• For instance, according to a study published in Science Direct, research was conducted to find out the patient preferences for home-based infusion therapy. The data from 30 patients suffering from Fabry, Gaucher, and Pompe Disease was analyzed, and it was demonstrated that home-based therapy increased the patient satisfaction and also imparted quality care compared to the therapy services provided in clinics or enzyme replacement therapy centers. Such scenarios lead to the growth of the market.

Enzyme Replacement Therapy Market Growth Factors

Increasing Prevalence of Rare Diseases to Drive Market Growth

There is a gradual increase in the global prevalence of different lysosomal storage disorders such as Gaucher, Fabry, Pompe, and MPS. For instance, according to the data published by the National Institute of Neurological Disorders and Stroke in August 2021, around 1 in 40,000 people in the U.S. were suffering from Pompe Disease,which is estimated to be around 32,950.

Also, according to the data published by Frontiers in January 2024, 11.6 million newborns were screened for Pompe Disease across 8 countries and 4 continents. A ratio of 1 to 18,711 new born were found to be suffering from the Pompe Disease. 

Also, according to the National Fabry foundation, in May 2020, the total Fabry patient population in the U.S. was around 7,713. Thus, the presence of a large patient pool suffering from rare lysosomal storage disorders generates higher demand for effective treatment options such as enzyme replacement therapy. Also, increasing initiatives by government and non-profit organizations in various countries to promote awareness among the general population lead to a gradual rise in the diagnosis rate of rare diseases. Hence, such key factors and favorable reimbursement policies and government funding for treatment in developed countries are increasing the adoption rate of this therapy, and subsequently, driving the market growth during the forecast period.

RESTRAINING FACTORS

High Cost of Treatment to Limit Market Growth

Availability of favorable reimbursement policies in developed countries and the rising prevalence of rare disorders are major factors for the market growth. However, the lack of skilled healthcare technicians for this therapy and inadequate reimbursement policies in emerging countries are some of the factors limiting the market growth.

• For instance, according to National Gaucher Foundation, the average cost for one Gaucher patient is around USD 0.2 million per year. Also, according to an article published by Elsevier B.V., it was stated that a significant delay was observed between the onset and initiation of the symptoms of the ERT process in the diseased population in Brazil, primarily due to lack of insurance coverage in this country.


• In September 2022, BioMed Central Ltd published data demonstrating that the cost-effectiveness ratio for Infantile-onset Pompe's disease was estimated at USD 1,130,039 per Quality-adjusted life year (QALY) when utilizing a dose of alglucosidase at 40 mg/kg per week. For adults with Pompe Disease, the reported ICER was USD 1.9 million/ QALY.

Also, the availability of alternative therapies, including chaperone therapy, in which pharmacological chaperones can easily cross the blood-brain barrier of the human body for effective treatment, leads to a higher preference for these therapies over the ERT procedures.

The above factors and various side effects, including pyrexia, flushing, dyspnoea, and others within the first 1-4 months of the therapy, further restrictit the global enzyme replacement therapy market growth.

Enzyme Replacement Therapy Market Segmentation Analysis

By Drug Class Analysis

Higher Adoption of Pancrelipase for the Treatment of EPI Led to its Dominant Position in 2023

Among drug class types, the pancrelipase segment accounted for the dominant position of the global enzyme replacement therapy market share in 2023. Rising prevalence of exocrine pancreatic insufficiency leads to higher demand and adoption rate of this drug class. Also, according to the annual report of key market players, such as AbbVie Inc. and others, it was estimated that this drug class is the highest contributing segment as compared to other ERT drugs classes, which leads to the dominance of this segment.

Additionally, collaborations between key market players to manufacture agalsidase are escalating the growth of the segment in the market.

• For instance, in February 2024, mAbxience announced the collaboration with Biosidus to manufacture the active ingredient Agalsidase Beta for the treatment of Fabry disease. Such collaborative efforts lead to the growth of the segment.

Additionally, the agalsidase segment held the second largest market share in 2023. The high cost and increasing demand for this drug for the treatment of Fabry disease are the major reasons for the significant market share of this segment.

By Route of Administration Analysis

Clinical Benefits Offered by Parenteral Route Poised to Surge the Demand during Forecast Period

Based on route of administration, the parenteral segment is expected to grow at the highest CAGR during the forecast period. This segment's dominance is attributed to the majority of drugs used in the therapy are available in the parenteral formulation.

The parenteral route offers several advantages, such as direct delivery into the bloodstream, bypassing gastrointestinal degradation, and consistent dosing, and it is also suitable for patients with gastrointestinal issues. Also, according to different research articles, parenteral administration of different drugs is highly effective compared to oral administration routes.

• For instance, according to Pharm approach Limited, the parenteral route of administration has several distinct advantages over the oral route such as high absorption rate, faster bioavailability, and others.

On the other hand, the oral segment captured a lower market share during the study period, owing to limited number of drugs available in oral formulation to treat key lysosomal storage disorders.

By Indication Analysis

Higher Prevalence of Exocrine Pancreatic Insufficiency Led to its Major Market Share in 2023

Based on indication, the exocrine pancreatic insufficiency segment captured the highest market share in 2023, owing to the growing prevalence of this disorder among general people. Also, the high occurrence of exocrine pancreatic insufficiency among patients with chronic pancreatitis and cystic fibrosis leads to an increase in the patient population of this disease. Thus, in turn, attributes to the dominance of this segment in 2023.

• For instance, according to the data published by the National Institute of Health (NIH) in January 2023, an estimated 42 to 73 per 100,000 of the population in the U.S. suffers from chronic pancreatitis, and 36 to 125 per 100,000 of the population suffers from chronic pancreatitis in Japan, China, and India. 60% to 90% of patients with chronic pancreatitis will suffer from exocrine pancreatic insufficiency within 10 to 12 years of diagnosis.

On the other hand, the others segment is expected to grow with the highest CAGR during the forecast period due to the emergence of new rare genetic and lysosomal disorders where ERT is the only preferable option.

• For instance, according to an article published by National Center for Biotechnology Information in November 2019, it was stated that Brineura, which is an enzyme replacement therapy, is the only treatment option for Neuronal ceroid lipofuscinosis type 2 disorder.

By End User Analysis

Growing Number of Patients Shift Toward Homecare Settings & Infusion Centers to Register the Highest CAGR

Based on End User, the homecare settings & infusion centers segment is expected to grow with the highest CAGR during the forecast period, owing to preferential shifts of patients diagnosed with rare diseases from hospital to home care settings. Also, different governments and private hospitals are encouraging patients for home care treatment during the COVID-19 pandemic by giving them proper training to administer the drugs independently, which is expected to boost the growth of this segment during the forecast period.

On the other hand, the hospital segment is expected to grow at a significant CAGR due to increasing number of hospitals in emerging countries, advanced medical equipment for infusion therapy, and the availability of skilled healthcare professionals for continuous use monitoring of patients.

REGIONAL INSIGHTS

The market size in North America stood at USD 6.45 billion in 2023. The rising prevalence of rare lysosomal storage disorders, such as Pompe Disease, Fabry Disease, and others, and the strong direct presence of key market players in this region are contributing factors to the dominance of this region in the global market. Additionally, the presence of many infusion centers in this region is augmenting the market growth.

• For instance, according to Healio, there were almost 3,600 infusion centers in the U.S. in 2019, catering to a large patient pool undergoing enzyme replacement therapy for various rare diseases.

Europe accounted for the second-largest share of the global market in 2023. The growth is primarily attributed to favorable reimbursement policies in a few European countries for different rare lysosomal disorders such as Gaucher disease, MPS, and others.

• For instance, according to F1000 Research Ltd., Polish National Health Fund launched a national drug program under which a patient received the first reimbursement in ERT for Fabry disease in 2019.

Also, rapid advancement in the healthcare infrastructure and increasing demand for treatment options for rare diseases fuel the growth of the market in this region. Asia Pacific is expected to grow at a significant CAGR during the forecast period. The growing number of patient populations suffering from rare disorders and increasing initiatives by the government and other private organizations for the awareness about this therapy are surging the demand and adoption of this therapy among general people and driving the market growth.

Latin America and the Middle East & Africa are expected to grow with a moderate CAGR during the forecast period due to limited number of infusion centers and lack of adequate reimbursement policies for treating rare diseases in these regions. However, governments and other private organizations are focusing on developing healthcare infrastructure in some countries, including Brazil, the UAE, Saudi Arabia, and others.

• For instance, according to a report by International Trade Administration in January 2021, it was stated that Brazil is the largest and most developing healthcare market in Latin America, spending around 9.1% of its GDP on healthcare.

Thus, increasing focus on the development of healthcare infrastructure and the rising prevalence of key lysosomal storage disorders are anticipated to surge the demand and adoption of ERT in the near future.

List of Key Companies in Enzyme Replacement Therapy Market

Wide Product Offerings by Key Players Led to Strong Market Presence

A few established players, such as BioMarin, Sanofi, AbbVie Inc., and Takeda Pharmaceutical Company Limited, dominate the market. A strong focus on acquisition and partnership with other major players of the market to expand the product reach globally is one of the key reasons for the market dominance of these companies.

• For instance, in September 2021, Takeda Pharmaceutical Company Limited announced a collaboration and partnership agreement with JCR Pharmaceuticals Co., Ltd., intending to commercialize JR-141 outside the U.S. to treatment of hunter syndrome.

On the other hand, emerging players, including TEIJIN LIMITED. Clinigen Group plc, and others are constantly focusing on new product launches for different therapeutic conditions. Also, a strong focus on receiving marketing approval from regulatory authorities is assisting them in catering to the rising demand for the treatment of lysosomal storage disorders in both developed and emerging countries.

For instance, in May 2019, TEIJIN LIMITED launched Revcovi 2.4 mg intending to treat Adenosine deaminase (ADA) deficiency through the parenteral route of administration.

The other key players operating in the market are Leadiant Biosciences, Inc., Pfizer Inc., Actelion Pharmaceuticals US, Inc., AstraZeneca, and Zoetis.

LIST OF KEY COMPANIES PROFILED:

• BioMarin (U.S.)


• Leadiant Biosciences, Inc. (U.S.)


• Pfizer Inc. (U.S.)


• Sanofi (France)


• AbbVie Inc. (U.S.)


• Takeda Pharmaceutical Company Limited Japan)


• JCR Pharmaceutical Co., Ltd. (Japan)


• Nestlé (Switzerland)

KEY INDUSTRY DEVELOPMENTS:

• November 2023- Takeda Pharmaceutical Company Limited announced the U.S. Food and Drug Administration (FDA) approval of ADZYNMA. It is the first and only recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy to treat adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).


• September 2023- Amicus Therapeutics announced the U.S. Food and Drug Administration approval and launch of Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) 65mg capsules to treat patients with late-onset Pompe disease.


• January 2021–Clinigen Group plc., announced the manufacturing and marketing approval for Hunterase ICV in Japan to treat patients with Hunter syndrome.


• October 2018– Leadiant Biosciences Inc., announced the U.S. FDA approval of Revcovi intending to treat adenosine deaminase, a severe immunodeficiency syndrome in pediatric and adult patients.

REPORT COVERAGE

The enzyme replacement therapy market report provides a detailed analysis of the industry and focuses on key aspects such as leading companies, products, and End Users. Besides this, it offers insights into the market trends and highlights key industry developments. In addition to the factors above, the market report encompasses several factors that have contributed to the growth of the advanced market over recent years.

Report Scope and Segmentation

ATTRIBUTE	DETAILS
Study Period	2019-2032
Base Year	2023
Estimated Year	2024
Forecast Period	2024-2032
Historical Period	2019-2022
Growth Rate	CAGR of  9.2% from 2024-2032
Unit	Value (USD Billion)
Segmentation	By Drug Class Alglucosidase alfa Agalsidase Pancrelipase Idursulfase Laronidase Imiglucerase Elosulfase alfa Asfotase alfa Galsulfase Velaglucerase alfa Others
By Route of Administration Parenteral Oral
By Indication Type 1 Gaucher disease Pompe Disease MPS (Mucopolysaccharidosis) Exocrine Pancreatic Insufficiency (EPI) Fabry Disease Hypophosphatasia Others
By End User Hospitals Homecare settings & Infusion Centers
By Geography North America (By Drug Class, By Route of Administration, By Indication, By End User, and By Country) U.S. (By Indication) Canada (By Indication) Europe (By Drug Class, By Route of Administration, By Indication, By End User, and By Country /Sub-region) U.K. (By Indication) Germany (By Indication) France (By Indication) Italy (By Indication) Spain (By Indication) Scandinavia (By Indication) Rest of Europe (By Indication) Asia Pacific (By Drug Class, By Route of Administration, By Indication, By End User, and By Country/Sub-region) Japan (By Indication) China (By Indication) India (By Indication) Australia (By Indication) Southeast Asia (By Indication) Rest of Asia Pacific (By Indication) Latin America (By Drug Class, By Route of Administration, By Indication, By End User, and By Country /Sub-region) Brazil (By Indication) Mexico (By Indication) Rest of Latin America (By Indication) Middle East& Africa (By Drug Class, By Route of Administration, By Indication, By End User, and By Country /Sub-region) South Africa (By Indication) GCC (By Indication) Rest of Middle East & Africa (By Indication)