The enzyme replacement therapy market is expected to grow from US$ 9,673.39 million in 2021 to US$ 15,184.70 million by 2028; it is estimated to grow at a CAGR of 6.8% from 2022 to 2028.

The report highlights trends prevailing in the market and factors driving the market growth. The growth of the global enzyme replacement therapy market is attributed to the rising prevalence of lysosomal storage diseases (LSDs) and rapid regulatory approval with other marketing benefits for drug with orphan drug designation. However, cutthroat competition among market players hinders the overall market growth.

Enzyme replacement therapy (ERT) is performed to treat congenital enzyme deficiencies by using enzyme or protein having enzymatic activity. Animal, human, and recombinant engineered enzymes are a few sources of enzyme required for treatment. The therapy is generally applied in rare and lysosomal storage diseases, including Pompe's disease, Fabry's disease, Gaucher's disease, Tay-Sachs disease, and Hurler syndrome. The most common ERT method is IV infusions, in which the replacement enzyme is administered directly into the bloodstream through a controlled drip of fluids.

European Medicines Agency (EMA) approves orphan drugs. For instance, according to EMA, if a product that has an orphan drug designation receives EMA marketing approval for the indication for which it has such designation, the product is entitled to orphan market exclusivity. It means the EMA may not approve any other application to market a similar drug for the same indication for 10 years.

Furthermore, in March 2016, the EMA launched the Priority Medicines ("PRIME") scheme to facilitate the development of product candidates in indications, often rare, for which few or no therapies currently exist. The PRIME scheme also provides several benefits to drug producers; once a candidate medicine is selected for PRIME. Below mentioned are the benefits provided by the EMA:

- Appoint a rapporteur from the Committee for Medicinal Products for Human Use (CHMP) and help to build knowledge ahead of a marketing-authorization application

- Early and proactive regulatory dialogue with the EMA to guide the overall development plan and regulatory strategy;

- Provide scientific advice at key development milestones, involving additional stakeholders such as health-technology-assessment bodies, to facilitate quicker access for patients to the new medicine;

- Confirm potential for accelerated assessment at the time of an application for marketing authorization.

Therefore, owing to the benefit and incentives associated with drugs designated as orphan drugs, key players operating in the market are putting efforts into developing drugs for orphan diseases. For instance, Amicus Therapeutics, Inc. recently obtained an orphan medicinal product designation in Europe from the EMA for Galafold to treat Fabry disease and the combination product, ATB200/AT2221, for treating Pompe disease. Additionally, in September 2020, Amicus Therapeutics, Inc. was granted PRIME designation for AT-GTX-501, therapy for CLN6 Batten disease.

Thus, the rapid regulatory approval with other marketing benefits for the drug with orphan drug designation is driving the market growth.

Based on enzyme type, the global enzyme replacement therapy market is segmented into alglucosidase alfa, agalsidase beta, imiglucerase, idursulfase, galsulfase, velaglucerase alfa, and other enzymes. In 2021, the other enzymes segment accounted for the largest market share. The same segment is expected to register the highest CAGR in the market from 2022 to 2028. Based on therapeutic conditions, the global enzyme replacement therapy market is segmented into Gaucher's disease, Fabry's disease, Pompe's disease, SCID, MPS, and other therapeutic conditions. The Gaucher's disease segment held the largest market share in 2021 and is expected to register the highest CAGR during the forecast period. Based on route of administration, the global enzyme replacement therapy market is bifurcated into parenteral and oral. The parenteral segment held a larger market share in 2021 and is anticipated to register a higher CAGR during the forecast period. Based on end user, the global enzyme replacement therapy market is segmented into hospitals, infusion centers, and others. The hospitals segment held the largest market share in 2021. However, the infusion centers segment is expected to register the highest CAGR during the forecast period.

Impact of COVID-19 Pandemic on Global Enzyme Replacement Therapy Market

The COVID-19 pandemic significantly affected the global healthcare system. To combat the spread of SAR-CoV-2 virus, governments imposed stringent policies to prevent and reduce the infection and mortality rates. According to the Department of Emergency Medicine, in many cities across various countries in North America, emergency department (ED) visits decreased by ~40% in 2020. Moreover, outpatient appointments and elective treatments were postponed or replaced by telemedicine practices.

Moreover, the interruption of ERT during the COVID-19 pandemic may have significant consequences. Due to restrictions on hospital visits during the pandemic, many scheduled ERTs were disrupted, which led to severity of conditions in LSDs patients. Also, lysosomal storage diseases are considered to be high risk because of respiratory, renal, and cardiac involvement. For instance, in 2021, a sample survey conducted by some doctors from Sir Ganga Ram Hospital, India, results of which were published in Indian Pediatrics, offered a glimpse into the crisis. The survey revealed that of the 26 patients suffering from lysosomal storage disorders and receiving ERT at the hospital, 20 (77%) had significant interruption in their treatment, with an average eight missed doses. Of the 20 patients who missed multiple doses, 13 (65%) could not collect the medicine from the tertiary care center due to lack of transportation as lockdown was in place, while for two (10%), the medicine import was delayed. Thus, such interruptions in ERT increased the suffering of LSDs patients and impacted the enzyme replacement therapy market negatively.

Various organic and inorganic strategies are adopted by companies operating in the global enzyme replacement therapy market. The organic strategies mainly include product launches and product approvals. Further, inorganic growth strategies witnessed in the market are acquisitions, collaborations, and partnerships. These growth strategies allow the market players to expand their businesses and enhance their geographic presence, thereby contributing to the overall market growth. Further, acquisition and partnership strategies help the market players in strengthening their customer base and expanding their product portfolios. A few of the significant developments by key players in the global enzyme replacement therapy market are listed below.

- In May 2022, AbbVie and Cugene Inc., a clinical-stage biotechnology company focused on developing next-generation precision immunology and oncology medicines to treat autoimmune disease and cancer, announced an exclusive worldwide license option agreement for CUG252, a potential best-in-class Treg-selective IL-2 mutein, as well as other novel IL-2 muteins, for the potential treatment of autoimmune and inflammatory diseases.

- In August 2021, the US Food and Drug Administration (FDA) approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients aged one year and above suffering from late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person's ability to move and breathe. Nexviazyme is an ERT designed to specifically target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of ERT in Pompe disease. Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance.

A few major primary and secondary sources referred to while preparing the report on the global enzyme replacement therapy market are the National Health and Medical Research Council (NHMRC), the Government of Canada, and the Bioprocessing Technology Institute (BTI).