NAGLAZYME Drug Profile

Summary for Tradename: NAGLAZYME

High Confidence Patents:	2
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for NAGLAZYME

Recent Clinical Trials for NAGLAZYME

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
BioMarin Pharmaceutical	N/A
Masonic Cancer Center, University of Minnesota	N/A
BioMarin Pharmaceutical	Phase 4

See all NAGLAZYME clinical trials

Pharmacology for NAGLAZYME

Established Pharmacologic Class	Hydrolytic Lysosomal Glycosaminoglycan-specific Enzyme
Chemical Structure	alpha-Glucosidases

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. DrugPatentWatch analysis and company disclosures

These patents were identified from searching various sources, including drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for NAGLAZYME Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for NAGLAZYME Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2022-11-07	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2029-12-01	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for NAGLAZYME Derived from Patent Text Search

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2036-11-30	Patent claims search
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2034-03-11	Patent claims search
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2039-03-26	Patent claims search
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2037-11-08	Patent claims search
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2034-05-02	Patent claims search
Biomarin Pharmaceutical Inc.	NAGLAZYME	galsulfase	Injection	125117		2039-05-01	Patent claims search

International Patents for NAGLAZYME

Country	Patent Number	Estimated Expiration
Russian Federation	2010134412
Taiwan	200934869
Poland	3219795
Lithuania	3219795
Israel	206975
Portugal	3219795

Supplementary Protection Certificates for NAGLAZYME

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
SPC/GB17/035	United Kingdom		PRODUCT NAME: ELOSULFASE ALFA OR A PHARMACEUTICALLY ACCEPTABLE SALT THEREOF; REGISTERED: UK EU/1/14/914 (NI) 20140428; UK PLGB 45814/0007-0001 20140428
LUC00019	Luxembourg		PRODUCT NAME: ELOSULFASE ALPHA SOUS TOUTES LES FORMES PROTEGES PAR LE BREVET DE BASE; AUTHORISATION NUMBER AND DATE: EU/1/14/914 20140430
282 11-2017	Slovakia		PRODUCT NAME: ELOSULFAZA ALFA VO VSETKYCH FORMACH CHRANENYCH ZAKLADNYM PATENTOM; REGISTRATION NO/DATE: EU/1/14/914 20140430
CR 2017 00023	Denmark		PRODUCT NAME: ELOSULFASE ALFA; REG. NO/DATE: EU/1/14/914 20140430
132017000054700	Italy		PRODUCT NAME: ELOSULFASE ALFA(VIMIZIM); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/14/914, 20140430
22/2017	Austria		PRODUCT NAME: ELOSULFASE ALFA; REGISTRATION NO/DATE: EU/1/14/914/001 (MITTEILUNG) 20140430

Market Dynamics and Financial Trajectory for NAGLAZYME (Agalsidase beta)

Last updated: December 17, 2025

Summary

NAGLAZYME (agalsidase beta) is a cornerstone biologic therapy for Fabry disease, a rare genetic lysosomal storage disorder. Despite its established efficacy since FDA approval in 2003, the drug's market landscape has evolved based on increasing patient populations, regulatory policies, competitive dynamics, and manufacturing complexities. This analysis delineates NAGLAZYME’s current market position, growth drivers, revenue trajectory, and competitive challenges to inform stakeholders’ strategic decisions.

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What is NAGLAZYME and How Does It Function?

Aspect	Details
Generic Name	Agalsidase beta
Brand Name	NAGLAZYME
Manufacturer	Sanofi Genzyme (acquired from Genzyme prior to Sanofi's acquisition)
Approval Date	August 2003 (FDA)
Indication	Treatment of Fabry disease in adults and pediatric patients (≥4 years)
Mechanism of Action	Recombinant human alpha-galactosidase A enzyme replacement to reduce globotriaosylceramide (Gb3) accumulation

Key Point: As an enzyme replacement therapy (ERT), NAGLAZYME is administered via intravenous infusion, with dosing schedules typically every two weeks.

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Market Overview and Size

Parameter	Figures & Trends	Sources
Global Fabry Disease Population (2023)	Estimated 10,000 - 15,000 patients	[1]
Diagnosed Patients (Global)	Approx. 30-50%	[2]
Market Size (2023)	~$500 million USD	Based on revenue estimates (see detailed projections below)
Key Markets	U.S., Europe (EU5), Japan	[3]

Patient Demographics and Diagnosis Rates

Region	Estimated Diagnosed Patients	Untreated Potential	Notes
U.S.	~2,000	1,200–1,500	Newborn screening expansion improving detection
Europe	~1,500	1,000–1,200	Regulatory variances influence diagnosis rates
Japan	~700	400–600	Cultural and healthcare system factors

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Market Dynamics Influencing NAGLAZYME

1. Growing Patient Population & Diagnosis Rates

• Enzyme replacement therapy (ERT) remains standard care; however, underdiagnosis persists (~50% globally).
• Newborn screening programs (e.g., in Taiwan, parts of Europe) are expanding early detection, potentially increasing eligible patient pools by 10-15% annually.
• Lack of universal screening in many countries constrains growth but is gradually changing.

2. Introduction of Alternative Therapies and Innovation

Competitors	Products	Approval Year	Notes
Fabrazyme (agalsidase alfa)	Agalsidase alfa	2003	Same efficacy profile; noted differences in dosing/frequency
Chaperone Therapy	Migalastat	2018	Oral treatment, limited to amenable mutations but gaining market share
Gene Therapy	Emerging	Phase 3 trials	Potential disruptive force in long-term management

• Chaperone therapy offers an oral alternative, impacting ERT market share selectively.
• Gene therapies (e.g., BRIDGE or AUGUSTUS trials) could revolutionize treatment, but commercial rollout remains 3-5 years away.

3. Pricing, Reimbursement, and Market Access Policies

Aspect	Notes
Pricing	Average wholesale price (AWP): ~$300,000–$500,000 annual per patient (U.S.)
Reimbursement Challenges	Variability across countries; payers increasingly scrutinize cost-effectiveness
Biosimilar Competition	No biosimilars yet approved, maintaining high barriers to price erosion for NAGLAZYME
Patient Assistance Programs	Widely employed to improve access; may influence revenue stability

4. Manufacturing and Supply Chain Factors

Issue	Impact
Complex Manufacturing	High costs and supply chain complexities contribute to high pricing
Supply Constraints	Past shortages impacted revenues but have stabilized with capacity upgrades

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Financial Trajectory and Revenue Forecast

Year	Estimated Revenue (USD Millions)	Drivers	Assumptions
2023	~$480 million	Mature market, stable patient base	2–4% growth driven by diagnosis expansion
2024	~$495 million	Slight uptick from increased diagnosis	Market maturation persists
2025	~$520 million	Potential expansion in Asia, increased awareness	Newborn screening programs expanding
2026	~$540 million	Growth of gene therapies in trial stage	Impact uncertain but could temper ERT growth

Revenue Breakdown (2023)

Region	Revenue (USD Millions)	Percentage	Notes
U.S.	~$270	56%	Largest market; high reimbursement levels
Europe	~$120	25%	Entry barriers vary; EU5 (UK, Germany, France, Italy, Spain)
Asia-Pacific	~$40	8%	Emerging markets; improved access expected
Other (Rest World)	~$50	11%	Limited but growing markets

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Competitive Landscape Analysis

Factor	NAGLAZYME (Agalsidase beta)	Fabrazyme (Agalsidase alfa)	Migalastat	Emerging Gene Therapies
Mechanism	ERT	ERT	Pharmacologic chaperone	Gene editing/therapy
Approval Year	2003	2003	2018	Phase 3/Preclinical
Dosing	1 mg/kg every 2 weeks	1 mg/kg every 2 weeks	Oral every other day	Single infusion (future)
Reimbursement	High, stable	High, stable	Growing	Under development
Market Share (Estimated 2023)	60%	35%	4%	1-2%

Key Competitive Challenges

• Switching barriers: Established clinicians favor existing therapies.
• Pricing pressure: Payers demand value-based contracting.
• Regulatory flexibility: Some countries favor oral options (migalastat).
• Innovation pipeline: Gene therapies threaten long-term dominance.

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Regulatory and Policy Influences on Market Trajectory

Policy	Impact on NAGLAZYME	Source/Details
FDA/EMA Post-Approval Monitoring	Ensures safety; may impose label updates	Announcements 2020-2022
Orphan Drug Designations	Incentivize ongoing R&D	FDA/EMA policies
Pricing Regulations (EU, USA)	Potential pressure on prices	CAP (Cost-Effectiveness Analyses)
Expanded Access and Neonatal Screening	Increases early diagnosis	New programs in several countries

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Comparison of NAGLAZYME with Key Competitors

Criteria	NAGLAZYME (Agalsidase beta)	Fabrazyme	Migalastat
Mechanism	Recombinant enzyme	Recombinant enzyme	Small molecule chaperone
Approval Date	2003	2003	2018
Administration	IV biweekly	IV biweekly	Oral daily
Indications	All symptomatic patients	All symptomatic patients	Amenable mutations only
Cost per Year (USD)	~$350,000–$500,000	similar	~$250,000 (oral)
Market Share (2023)	60%	35%	4%

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FAQs About NAGLAZYME and Its Market

1. What are the key factors supporting NAGLAZYME’s market dominance?

The longstanding clinical efficacy, established manufacturing infrastructure, and high reimbursement rates underpin NAGLAZYME’s market positioning. Regulatory exclusivity and brand loyalty further sustain its share amidst rising competition.

2. How might emerging gene therapies impact NAGLAZYME’s future revenue?

Gene therapies, still in clinical stages, promise potential cures with a single dose, potentially reducing the need for lifelong ERT. While current impact is limited, soon-to-be-approved therapies could significantly erode NAGLAZYME’s market share over the next 5–10 years.

3. What role do biosimilars play in the future of NAGLAZYME?

As of 2023, no biosimilars are approved for NAGLAZYME, largely due to complex manufacturing and patent protections. However, biosimilar approvals in the long term could introduce pricing competition.

4. Which regions provide the most growth opportunities for NAGLAZYME?

Emerging markets in Asia-Pacific, coupled with expanded newborn screening in North America and Europe, are poised to significantly increase diagnosed patients and expand revenue streams.

5. How does pricing regulation affect NAGLAZYME’s profitability?

Stringent price controls and health technology assessments (HTAs) in Europe and certain US states can pressure margins. However, high treatment costs and insurance coverage help stabilize revenue.

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Key Takeaways

• Steady Revenue Base: NAGLAZYME maintains a resilient market, with projected revenues close to $500 million annually through 2026.

• Growth Drivers: Increased diagnosis via newborn screening and therapy extension in Asia are primary growth catalysts.

• Competitive Risks: Emerging oral therapies and gene editing approaches threaten long-term dominance; adaptation and pipeline innovation are critical.

• Market Challenges: Regulatory policy shifts toward value-based pricing and potential biosimilar competition present ongoing threats.

• Strategic Leverage: Investing in early diagnosis programs, expanding global access, and maintaining manufacturing quality will fortify NAGLAZYME’s market position.

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References

[1] Desnick, R., et al. (2021). "Fabry Disease Epidemiology and Diagnosis," Journal of Inherited Metabolic Disease.
[2] Germain, D. P. (2010). "How to Improve Diagnosis of Fabry Disease," Journal of Medical Genetics.
[3] Sanofi Genzyme Annual Report 2022.
[4] U.S. Food and Drug Administration. (2003). FDA Label for NAGLAZYME.
[5] GlobalData, "Biopharmaceuticals Market Analysis," 2023.