North America Enzyme Replacement Therapy Market Forecast to 2028 – COVID-19 Impact and Regional Analysis – by Enzyme Type (Alglucosidase Alfa, Agalsidase Beta, Imiglucerase, Idursulfase, Galsulfase, Velaglucerase Alfa, and Other Enzymes), Therapeutic Conditions (Gaucher's Disease, Fabry's Disease, MPS, Pompe's Disease, SCID, and Other Therapeutic Conditions), Route of Administration (Parenteral and Oral), and End User (Hospitals, Infusion Centers, and Others)

The enzyme replacement therapy market in North America is expected to grow from US$ 4,108.69 million in 2022 to US$ 6,120.01 million by 2028; it is estimated to grow at a CAGR of 6.9% from 2022 to 2028.

Rapid Regulatory Approval with Other Benefits for Drugs with Orphan Drug Designation

A condition that affects fewer than 200,000 people nationwide is termed as orphan disease. Fabry's disease, Pompe disease, and Hunter syndrome are among a few examples. Supporting the development and evaluation of new therapies for rare diseases is a key priority for the Food and Drug Administration (FDA). The FDA has the authority to grant orphan drug designation to a drug or biological product. Additionally, stakeholders involved in the development of orphan drug molecules are eligible for the following benefits:

• 7-year marketing exclusivity is provided to sponsors of approved orphan products
• 25% federal tax credit for expenses is incurred in conducting clinical research within the US
• Tax credits may be applied to the prior year or applied over as many as 20 years to future taxes
• Prescription Drug User Fee Act (PDUFA) waivers fee for orphan drugs
• Ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for orphan drugs
• Eligibility to receive regulatory assistance and guidance from the FDA in designing an overall drug development plan.

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

Market Overview

The North America enzyme replacement therapy market is segmented into the US, Canada, and Mexico. The US enzyme replacement therapy market holds the largest share of the market. The market growth is due to the rise in the prevalence of lysosomal storage diseases and increasing regulatory approval of enzyme replacement therapy products. Intravenous (IV) infusions are used in enzyme replacement therapy (ERT) to treat the underlying enzyme shortage that underlies rare diseases, including Gaucher, Fabry's, and Hunter's syndrome. With the help of a modified form of the enzyme, ERT balances low levels of the glucocerebrosidase (GCase) enzyme, which builds up in the bodies of patients with Gaucher, Hunter, and Fabry's disease and is broken down by this enzyme. Therefore, rising cases of these rare diseases will require the need of developing novel treatments. For instance, according to the National Organization for Rare Disorders (NORD), the number of people in the US who had Gaucher disease was ~6,000 in  2021. According to statistics released by the National Institute of Neurological Disorders and Stroke in August 2021, there are an estimated 32,950 cases of Pompe disease in the US, affecting 1 in 40,000 persons. The National Fabry Foundation estimates that there were ~7,713 Fabry patients in the US as of May 2020. Thus, a restricted number of rare diseases are treated through enzyme replacement therapies, which is boosting the market growth across the region.

 North America Enzyme Replacement Therapy Market Revenue and Forecast to 2028 (US$ Million)

North America Enzyme Replacement Therapy Market Segmentation 

The North America enzyme replacement therapy market is segmented into enzyme type, therapeutic conditions, route of administration, end user, and country.

• Based on enzyme type, the North America enzyme replacement therapy market is segmented into alglucosidase alfa, agalsidase beta, imiglucerase, idursulfase, galsulfase, velaglucerase alfa, and other enzymes. The other enzymes segment registered the largest market share in 2022.
• By therapeutic conditions, the North America enzyme replacement therapy market is segmented into Gaucher's disease, Fabry's disease, MPS, Pompe's disease, SCID, and other therapeutic conditions. The Gaucher's disease segment held the largest market share in 2022.
• In terms of route of administration, the North America enzyme replacement therapy market is bifurcated into parenteral and oral. The parenteral segment held a larger market share in 2022.
• From end user point of reference, the North America enzyme replacement therapy market is segmented into hospitals, infusion centers, and others. The hospitals segment held the largest market share in 2022.
• Based on country, the North America enzyme replacement therapy market is segmented into the US, Canada, and Mexico. The US dominated the market in 2022.

Sanofi; BioMarine Pharmaceutical Inc; Takeda Pharmaceutical Company Limited; AbbVie Inc; Janssen Pharmaceutical (Johnson & Johnson Services, Inc.); Alexion Pharmaceutical, Inc. (AstaZeneca); Amicus Therapeutics; Recordati S.p.A; Recordati S.p.A; CHIESI farmaceutici S.p.A; and Pfizer Inc are the leading companies operating in the enzyme replacement therapy market in North America.   

1. Sanofi.
2. BioMarine Pharmaceutical Inc.
3. Takeda Pharmaceutical Company Limited.
4. AbbVie Inc.
5. Janssen Pharmaceutical ( Johnson & Johnson Services,Inc.).
6. Alexion Pharmaceutical, Inc.(AstaZeneca).
7. Amicus Therapeutics.
8. Recordati S.p.A.
9. CHIESI farmaceutici S.p.A.
10. Pfizer Inc.

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