VIMIZIM Drug Profile

Summary for Tradename: VIMIZIM

High Confidence Patents:	10
Applicants:	1
BLAs:	1
Recent Clinical Trials:	See clinical trials for VIMIZIM

Recent Clinical Trials for VIMIZIM

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
ICON plc
BioMarin Pharmaceutical

See all VIMIZIM clinical trials

Pharmacology for VIMIZIM

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 VIMIZIM Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for VIMIZIM 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.	VIMIZIM	elosulfase alfa	Injection	125460	10,100,311	2036-07-29	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	10,240,149	2035-06-02	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	10,544,426	2031-10-13	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	11,180,749	2039-08-07	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	8,236,315	2029-01-23	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	8,277,813	2027-10-31	DrugPatentWatch analysis and company disclosures
Biomarin Pharmaceutical Inc.	VIMIZIM	elosulfase alfa	Injection	125460	8,586,036	2030-04-09	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for VIMIZIM 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.	VIMIZIM	elosulfase alfa	Injection	125460	9,474,688	2032-10-25	Patent claims search

International Patents for VIMIZIM

Country	Patent Number	Estimated Expiration
Austria	E524548
Mexico	394708
World Intellectual Property Organization (WIPO)	2012049266
China	110249059
Lithuania	2627183
Spain	2495092
Russian Federation	2615143

Supplementary Protection Certificates for VIMIZIM

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2017024	Norway		PRODUCT NAME: ELOSULFASE ALFA ELLER ET FARMASOEYTISK AKSEPTABELT SALT DERAV; REG. NO/DATE: EU/1/14/914 20140523
CR 2017 00023	Denmark		PRODUCT NAME: ELOSULFASE ALFA; REG. NO/DATE: EU/1/14/914 20140430
122017000036	Germany		PRODUCT NAME: ELOSULFASE ALFA IN ALLEN SEINEN DURCH DAS GRUNDPATENT GESCHUETZTEN FORMEN; REGISTRATION NO/DATE: EU/1/14/914 20140428
CA 2017 00023	Denmark		PRODUCT NAME: ELOSULFASE ALFA IN ALL FORMS PROTECTED BY THE BASIC PATENT; REG. NO/DATE: EU/1/14/914 20140430
2017/020	Ireland		PRODUCT NAME: ELOSULFASE ALFA IN ALL FORMS PROTECTED BY THE BASIC PATENT; REGISTRATION NO/DATE: EU/1/14/914 20140428
C201730020	Spain		PRODUCT NAME: ELOSULFASE ALFA EN TODAS LAS FORMAS PROTEGIDAS POR LA PATENTE BASE; NATIONAL AUTHORISATION NUMBER: EU/1/14/914; DATE OF AUTHORISATION: 20140428; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/14/914; DATE OF FIRST AUTHORISATION IN EEA: 20140428
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

Market Dynamics and Financial Trajectory for VIMIZIM

Last updated: February 20, 2026

What is the current market position of VIMIZIM?

VIMIZIM (elosulfase alfa) is a biologic enzyme replacement therapy approved for treating mucopolysaccharidosis type IVA (Morquio A syndrome). It was developed by Ultragenyx Pharmaceutical and approved by the FDA in February 2014. The global market for Morquio A syndrome therapies remains limited due to the rarity of the disease, with VIMIZIM being the only FDA-approved biologic for this indication.

• Market Size (2023): Estimated global Morquio A population: 1,000 to 2,000 patients.
• Pricing: U.S. list price approximates $375,000 annually per patient.
• Sales (2022): Approximately $122 million, representing growth driven by increasing awareness and diagnosis rates.

How do market dynamics influence VIMIZIM's growth?

Key drivers:

• Rare disease diagnosis rates: Improved detection methods increase diagnosed cases, expanding the treatment pool.
• Pricing and reimbursement: High costs foster reliance on payor negotiations; payors are increasingly reluctant to reimburse at the initial list price.
• Competitive landscape: Limited direct competitors; other enzyme replacement therapies (ERTs), such as GALNS-based therapies (e.g., ZUPLENZA), target similar MPS types but not Morquio A specifically.
• Treatment access and administration: Intravenous infusion protocols limit outpatient adoption, influencing market penetration.

Limiters:

• Small patient population: Market expansion is constrained by disease rarity.
• Treatment burden: Weekly infusions and infusion-related adverse events reduce patient acceptance.
• Pricing pressure: Payers scrutinize high-cost therapies, leading to managed entry agreements, discounts, and potential biosimilar threats.

What are the financial trends impacting VIMIZIM?

Revenue growth trajectory:

Year	Global Sales (USD million)	Remarks
2018	95	Expansion of approved indications in Europe.
2019	105	Launch in additional markets; increased diagnosis.
2020	111	COVID-19 pandemic slowed infusion schedules.
2021	116	Continued global hospital outreach.
2022	122	Steady growth; price adjustments and expanded access.

Cost considerations:

• Manufacturing: Complex biologic production increases costs.
• Research & Development: Ongoing efforts to develop next-generation therapies.
• Regulatory expenses: Approvals in multiple jurisdictions incur high costs.

Outlook and projections:

• Compound annual growth rate (CAGR): Estimated at 4-6% through 2027, driven by diagnosis rate improvements and potential label extensions.
• Market risk factors: New therapies targeting Morquio A, such as gene therapies, could disrupt current revenue streams.
• Potential for label expansion: Clinical trials exploring use in other MPS types or pediatric populations may increase the market size.

How might external factors impact VIMIZIM's financial trajectory?

• Regulatory developments: Accelerated approval pathways in the U.S. and Europe could boost sales.
• Reimbursement policies: Stricter cost controls could pressure margins.
• Pricing reforms: Global shifts toward value-based pricing models could limit revenue growth.
• Advances in gene therapy: Successful development of gene-based treatments may replace ERT options, impacting future sales.

What are the strategic implications for stakeholders?

• Manufacturers: Focus on improving treatment convenience, reducing costs, and expanding indications.
• Investors: Monitor pipeline progress and entry of rival therapies.
• Healthcare providers: Evaluate cost-effectiveness and access issues.
• Regulators: Balance access with cost containment, influencing pricing policies.

Key Takeaways

• VIMIZIM has a limited but steady market constrained by disease rarity.
• Revenue growth remains positive, supported by increased diagnosis and broader market access.
• Cost structure is high due to complex biologic manufacturing and R&D.
• External factors, including regulatory changes and potential competition, bear influence on long-term financial prospects.
• Strategic focus on indication expansion, treatment delivery, and cost management will be critical.

FAQs

1. What is the primary indication for VIMIZIM?

VIMIZIM is approved for treating Morquio A syndrome, a rare lysosomal storage disorder caused by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase.

2. How does VIMIZIM compare pricing-wise with other enzyme replacement therapies?

VIMIZIM’s annual list price approximates $375,000 per patient. This aligns with other industry-standard RRTs for rare diseases but faces increasing payor scrutiny due to high costs.

3. Are there any upcoming regulatory decisions affecting VIMIZIM?

Likely, ongoing trials for potential label extensions and regulatory pathways in Europe and key Asian markets could influence future sales. No immediate approvals are scheduled, but the pipeline remains active.

4. What market risks could threaten VIMIZIM’s commercial performance?

Emergence of gene therapies for Morquio A could render enzyme replacement less attractive. Additionally, pricing constraints and biosimilar entry if patents lapse may impact revenues.

5. What strategies could maximize VIMIZIM’s future revenue?

Expanding indication approvals, optimizing infusion protocols, improving patient access, and engaging in value-based pricing negotiations could enhance long-term sales growth.

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References

1. Ultragenyx Pharmaceutical Inc. (2023). VIMIZIM (elosulfase alfa) product information. Retrieved from [Ultragenyx official site].
2. EvaluatePharma. (2023). World market for rare disease biologics. Retrieved from [EvaluatePharma database].
3. U.S. Food and Drug Administration. (2014). VIMIZIM approval announcement. Retrieved from [FDA site].
4. IQVIA. (2022). Biologic drug sales analysis. Retrieved from [IQVIA Reports].
5. European Medicines Agency. (2022). Marketing authorization updates for lysosomal storage disorders. Retrieved from [EMA site].

[1] Ultragenyx Pharmaceutical Inc. (2023). VIMIZIM (elosulfase alfa). Clinical data and product information.