Enzyme Replacement Therapy Market 2025 Growth & Forecast

The global enzyme replacement therapy market was valued at USD 13.45 billion in 2023 and is projected to grow from USD 14.61 billion in 2024 to USD 29.49 billion by 2032, at a compound annual growth rate (CAGR) of 9.2% during the forecast period. North America led the market in 2023, holding a 47.96% share.

Enzyme replacement therapy involves the administration of specific enzymes to patients with enzyme deficiencies or malfunctions. The increasing prevalence of rare, chronic, and inherited disorders, such as lysosomal storage diseases and genetic conditions, is driving the demand for effective treatment options. According to the Cleveland Clinic, approximately 6,000 individuals in the U.S. had Gaucher disease in 2020, with 95% diagnosed with type 1 Gaucher disease.

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List Of Key Companies Profiled in Enzyme Replacement Therapy Market:

• 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)

Enzyme Replacement Therapy Market Segmentation:

By Drug Class Analysis

Pancrelipase Dominates Due to High Demand in Exocrine Pancreatic Insufficiency (EPI) Treatment

In 2023, the pancrelipase segment held the largest share in the enzyme replacement therapy market, driven by the rising prevalence of exocrine pancreatic insufficiency (EPI). According to annual reports from leading companies like AbbVie Inc., pancrelipase is the highest-contributing drug class in the enzyme replacement therapy sector. The agalsidase segment held the second-largest share, with demand fueled by its effectiveness in treating Fabry disease. The market is further stimulated by strategic partnerships. For instance, in February 2024, mAbxience partnered with Biosidus to manufacture agalsidase beta, further driving segment growth.

By Route of Administration

Parenteral Administration Expected to Lead Market Growth

The parenteral segment is projected to experience the highest CAGR during the forecast period, as most enzyme replacement therapy drugs are formulated for parenteral use. This route offers advantages such as direct bloodstream delivery, bypassing gastrointestinal degradation, and ensuring consistent dosing. Research from Pharm Approach Limited highlights the superiority of parenteral administration in terms of absorption rate and bioavailability. In contrast, the oral segment holds a smaller market share due to the limited availability of oral enzyme replacement therapy formulations for treating lysosomal storage disorders.

By Indication

Exocrine Pancreatic Insufficiency (EPI) Holds the Largest Market Share

The exocrine pancreatic insufficiency segment dominated the market in 2023, driven by its high prevalence among patients with chronic pancreatitis and cystic fibrosis. According to National Institutes of Health (NIH) data (January 2023), chronic pancreatitis affects between 42 to 73 per 100,000 people in the U.S., with even higher prevalence rates in Japan, China, and India.

Over 60% to 90% of chronic pancreatitis patients develop EPI within 10 to 12 years of diagnosis, increasing demand for enzyme replacement therapy. Meanwhile, the "others" segment is expected to register the highest CAGR due to emerging rare lysosomal and genetic disorders requiring enzyme replacement therapy For instance, National Center for Biotechnology Information (November 2019) noted that Brineura is the only available treatment for Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disorder, further expanding the scope of enzyme replacement therapy applications.

By End User

Shift Toward Homecare Settings & Infusion Centers Expected to Drive Growth

The homecare settings and infusion centers segment is forecasted to witness the highest CAGR, fueled by the increasing preference for at-home treatment among patients with rare diseases. The COVID-19 pandemic accelerated this trend, with hospitals and governments encouraging home-based care by providing training for self-administration of enzyme replacement therapy. Conversely, the hospital segment is anticipated to grow at a substantial rate due to the rising number of hospitals in developing nations, the availability of advanced infusion equipment, and access to skilled healthcare professionals for patient monitoring.

Enzyme Replacement Therapy Market Growth Factors

Rising Prevalence of Rare Diseases

The increasing incidence of lysosomal storage disorders like Gaucher, Fabry, Pompe, and MPS is a key driver of market growth. As per the National Institute of Neurological Disorders and Stroke (August 2021), Pompe disease affects around 1 in 40,000 people in the U.S., translating to approximately 32,950 individuals. Additionally, a study published in Frontiers (January 2024) reported that 11.6 million newborns across eight countries were screened for Pompe disease, revealing a prevalence rate of 1 in 18,711 newborns. Similarly, the National Fabry Foundation estimated that 7,713 individuals in the U.S. had Fabry disease as of May 2020. The increasing patient pool and enhanced awareness campaigns by governments and non-profit organizations are expected to boost the diagnosis and adoption of enzyme replacement therapy. Favorable reimbursement policies and government funding in developed regions also support market expansion.

Enzyme Replacement Therapy Market Restraints

High Cost of Treatment

Despite the availability of reimbursement policies in developed countries, the high cost of enzyme replacement therapy remains a significant challenge. Emerging markets, in particular, face reimbursement limitations and a shortage of skilled healthcare professionals for ENZYME REPLACEMENT THERAPY administration. According to the National Gaucher Foundation, the annual cost for treating one Gaucher patient is approximately USD 200,000. Moreover, a BioMed Central Ltd study (September 2022) indicated that the cost-effectiveness ratio for treating infantile-onset Pompe disease was USD 1,130,039 per quality-adjusted life year (QALY) at a dose of 40 mg/kg per week. For adults, the incremental cost-effectiveness ratio (ICER) was USD 1.9 million per QALY.

The availability of alternative therapies, such as chaperone therapy, which can cross the blood-brain barrier for more effective treatment, also poses a competitive challenge. Furthermore, ENZYME REPLACEMENT THERAPY-related side effects, including pyrexia, flushing, and dyspnea, typically observed within the first one to four months of treatment, may hinder market adoption.

Key Industry Developments:

• November 2023 – Takeda Pharmaceutical Company Limited received FDA approval for ADZYNMA, the first and only recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy designed for the treatment of both adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

• September 2023 – Amicus Therapeutics secured FDA approval and launched Pombiliti (cipaglucosidase alfa-atga) in combination with Opfolda (miglustat) 65mg capsules for the treatment of patients with late-onset Pompe disease.