The market of Enzyme Replacement Therapy Market in the USA is growing since there is early detection of rare lysosomal storage disorders, extensive newborn screening, and increasing health care expenditure that forces patients towards biologics therapy. Enzyme replacement therapy remains the standard treatment for disorders like Gaucher, Fabry, and Pompe diseases, with the growing support from government institutions for further advancements in development of advanced therapies.
According to the SNS Insider report, the US market of enzyme replacement therapy reached the value of USD 4.26 billion in 2025 and is estimated to reach USD 9.30 billion in 2035, with CAGR of 8.12% due to patient-centric approach, reimbursement opportunities, and increased awareness of physicians and public for rare diseases, which leads to earlier diagnosis. This is on top of the global enzyme replacement therapy market valued at USD 12.24 billion in 2025 and estimated to reach USD 27.90 billion by 2035 at CAGR of 8.59% due to extended newborn screening and increasing orphan drugs incentives.
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Why the U.S. Leads Global Demand
United States was the largest contributor to North American enzyme replacement therapy revenue, due to the presence of highly advanced medical technology, well developed research and development framework, and sufficient healthcare budget to maintain an adequate stock of treatment facilities. Insurance coverage remains a major factor in making such expensive treatments available to more people.
The North America market represented 38.76% share of the total global market in 2025, of which the United States comprised around 90%. The presence of key players such as Pfizer, Takeda, and Sanofi continues to drive innovation and the accessibility of such treatments in the region.
Regulatory Momentum Is Accelerating the Pipeline
DNL310 (tividenofusp alfa) developed by Denali Therapeutics received Breakthrough Therapy Designation from the FDA for Hunter syndrome in January 2025, which is an important regulatory milestone for next generation enzyme replacement therapy that is now being tested through the pipeline in the United States. This also continues the trend of FDA involvement with the therapeutic class, which also includes Fast Track designation of GC1130A for Sanfilippo syndrome type A by GC Biopharma in 2024.
Recently, there was a multicenter newborn screening pilot study for five common lysosomal storage diseases, during which around 65,000 babies were screened, resulting in 69 initial positive results.
Segment Trends Shaping the U.S. Market
Imiglucerase was the dominant product type, accounting for about 22.8% market share in 2025, owing to its long-term success and consistency of clinical outcomes making it the ideal natural first-line treatment for Gaucher disease. The fastest-growing product type was agalsidase beta, spurred by increasing diagnoses of Fabry disease and access expansion programs by manufacturers.
Gaucher disease accounted for the highest demand among therapeutic conditions at 30.25% market share, whereas MPS was the fastest-growing therapy due to advances in diagnosis technology that helped identify patients with MPS disease at an early age. Parenteral administration was the leading mode of delivery, accounting for 83.10% market share, although the oral formulations were the fastest-growing modes as technological advances in enzyme encapsulation and protective coatings help address digestive tract degradation problems.
Companies Driving Innovation in the U.S. Market
Denali Therapeutics
FDA approval of the breakthrough therapy designation for DNL310 (tividenofusp alfa), a drug to treat Hunter syndrome, was awarded to Denali Therapeutics in January 2025, placing the company on the cutting edge of the next generation of ERT treatments. The breakthrough therapy designation will speed up the regulatory process for this rare disease that lacks available treatment options in the United States.
Takeda Pharmaceutical Company
ADZYNMA (ADAMTS13, recombinant-krhn), developed by Takeda, was approved by the FDA in 2023 for congenital thrombotic thrombocytopenic purpura. It is the first and only FDA-approved recombinant ADAMTS13 protein, adding to Takeda’s reputation as a leader in the U.S. enzyme therapy market.
Pfizer Inc.
BEQVEZ (fidanacogene elaparvovec-dzkt) received FDA approval from Pfizer in 2024, and this is a gene therapy product that is designed for use by adults who suffer from moderate to severe hemophilia B. The approval of BEQVEZ highlights Pfizer’s commitment towards developing biologic therapies for the treatment of rare genetic disorders.
Other established players active in the U.S. include Sanofi, Amicus Therapeutics, BioMarin Pharmaceutical, and Alexion Pharmaceuticals, each maintaining significant rare disease treatment portfolios.
Market Dynamics
Growth drivers of increasing prevalence and diagnosis of rare lysosomal storage diseases have been attributed to increased number of genetic tests being performed as well as newborn screening procedures, which help to detect rare diseases at a younger age. High cost of treatment will be the key limitation for this industry, considering high annual per patient treatment costs that often reach hundreds of thousands of dollars.
Opportunities in this industry include growth of orphan drugs pipelines due to FDA programs, such as tax breaks and market exclusivities, which attract pharmaceutical and biotechnology companies to enter the rare diseases market.
The Future of the U.S. Enzyme Replacement Therapy Market
The market in the United States is set up for long-term growth until 2035 due to increasing newborn screening adoption, ongoing FDA support for orphan drugs, and increased research and development into easy-to-administer oral drug formulations that require less frequent dosing. Organizations working on the development of new delivery systems and patient identification via diagnostics would be best positioned to benefit from growth in this space.
