Enzyme Replacement Therapy Market Size, Share & COVID-19 Impact Analysis, By Drug Class (Alglucosidasealfa, Agalsidase, Pancrelipase, Idursulfase, Laronidase, Imiglucerase, Elosulfasealfa, Asfotasealfa, Galsulfase, Velaglucerasealfa, 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 2021-2028

The global enzyme replacement therapy market size was USD 10.56 billion in 2020. The market is projected to grow from USD 11.43 billion in 2021 to USD 20.58 billion in 2028 at a CAGR of 8.8% during the 2021-2028 period. The global impact of COVID-19 has been unprecedented and staggering, with enzyme replacement therapy witnessing a negative impact on demand across all regions amid the pandemic. Based on our analysis, the global market exhibited a lower growth of 4.7% in 2020 as compared to the average year-on-year growth during 2017-2019.  The rise in CAGR is attributable to this market’s demand and growth, returning to pre-pandemic levels once the pandemic is over.

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.

COVID-19 IMPACT

Increased Demand for Enzyme Replacement Therapy during Pandemic to Augment Market Growth

The COVID-19 pandemic has positively impacted the global market. Major players operating in this market, including BioMarin, Sanofi, AbbVie Inc., and Takeda Pharmaceutical Company Limited. witnessed a huge growth in the sales of drugs used in this therapy.

• For instance, BioMarin reported a growth of 14.6% from the revenue of drugs used in this ERT in 2020. Also, AbbVie Inc., reported a growth of 7% from the revenue of CREON used in the treatment of exocrine pancreatic insufficiency in 2020 compared to 2019.

Also, safety precautions and guidelines followed in various healthcare settings, including hospitals and infusion centers resulted in the continuity of this treatment procedure during the pandemic. Furthermore, increasing initiatives by healthcare professionals to provide training sessions to patients for self-administration prevented delays in treatment during the COVID-19 pandemic.

• For instance, according to a report published by Oxford University Press, the enzyme replacement process was continued in the pandemic for Fabry disease in specific secure healthcare settings. Also, it was reported that treatment in these settings was beneficial to overcome the risk of developing COVID-19 due to safety guidelines.

Thus, several initiatives by healthcare authorities and the introduction of new reimbursement policies by the governments of developed countries to provide affordable infusion products for the patients at home impacted the global market positively during the COVID-19 pandemic.

LATEST TRENDS

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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.

DRIVING 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 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 thegeneral 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.

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.

SEGMENTATION

By Drug Class Analysis

Higher Adoption of Pancrelipase for the Treatment of EPI Led to its Dominant Position in 2020

Among drug class types, the pancrelipase segment accounted for the dominant share in 2020. 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, the agalsidase segment holds the second largest market share in 2020. 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. 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

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Higher Prevalence of Exocrine Pancreatic Insufficiency Led to its Major Market Share in 2020

Based on indication, the exocrine pancreatic insufficiency segment captured the highest market share in 2020, 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 2020.

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

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The market size in North America stood at USD 5.09 billion in 2020. 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 2020. 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.

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• 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.

KEY INDUSTRY PLAYERS

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 (San Rafael, U.S.)


• Leadiant Biosciences, Inc. (Gaithersburg, U.S.)


• Pfizer Inc. (New York, U.S.)


• Sanofi (Paris, France)


• AbbVie Inc. (Lake Bluff, U.S.)


• Takeda Pharmaceutical Company Limited (Tokyo, Japan)


• JCR Pharmaceutical Co., Ltd. (Ashiya, Japan)


• Nestlé (Vevey, Switzerland)

KEY INDUSTRY DEVELOPMENTS:

• 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

An Infographic Representation of Enzyme Replacement Therapy Market

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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.

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