VOXZOGO Drug Patent Profile

When do Voxzogo patents expire, and what generic alternatives are available?

Voxzogo is a drug marketed by Biomarin Pharm and is included in one NDA. There are seven patents protecting this drug and one Paragraph IV challenge.

This drug has one hundred and four patent family members in thirty countries.

The generic ingredient in VOXZOGO is vosoritide. One supplier is listed for this compound. Additional details are available on the vosoritide profile page.

DrugPatentWatch^® Generic Entry Outlook for Voxzogo

Voxzogo was eligible for patent challenges on November 19, 2025.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be October 20, 2030. This may change due to patent challenges or generic licensing.

There is one Paragraph IV patent challenge for this drug. This may lead to patent invalidation or a license for generic production.

Indicators of Generic Entry

Summary for VOXZOGO

International Patents:	104
US Patents:	7
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	2
Patent Applications:	87
Drug Prices:	Drug price information for VOXZOGO
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for VOXZOGO
DailyMed Link:	VOXZOGO at DailyMed

See drug prices for VOXZOGO

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for VOXZOGO

Constraining patent/regulatory exclusivity:

TO INCREASE LINEAR GROWTH IN PEDIATRIC PATIENTS LESS THAN 5 YEARS OF AGE WITH ACHONDROPLASIA WITH OPEN EPIPHYSES

NDA:

214938

Dosage:

POWDER;SUBCUTANEOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for VOXZOGO

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Roopa Kanakatti Shankar	Phase 2
UCSF Benioff Children's Hospital Oakland	Phase 1/Phase 2

See all VOXZOGO clinical trials

Paragraph IV (Patent) Challenges for VOXZOGO

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
VOXZOGO	Powder for Injection	vosoritide	0.4 mg/vial 0.56 mg/vial 1.2 mg/vial	214938	1	2025-11-19

US Patents and Regulatory Information for VOXZOGO

VOXZOGO is protected by seven US patents and four FDA Regulatory Exclusivities.

This potential generic entry date is based on TO INCREASE LINEAR GROWTH IN PEDIATRIC PATIENTS LESS THAN 5 YEARS OF AGE WITH ACHONDROPLASIA WITH OPEN EPIPHYSES.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes			Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes

EU/EMA Drug Approvals for VOXZOGO

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
BioMarin International Limited	Voxzogo	vosoritide	EMEA/H/C/005475Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.	Authorised	no	no	yes	2021-08-26

International Patents for VOXZOGO

See the table below for patents covering VOXZOGO around the world.

Country	Patent Number	Title	Estimated Expiration
China	117881416
Hong Kong	1255864
World Intellectual Property Organization (WIPO)	2023283657

Supplementary Protection Certificates for VOXZOGO

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2432489	C202230004	Spain		PRODUCT NAME: VOSORITIDE; NATIONAL AUTHORISATION NUMBER: EU/1/21/1577; DATE OF AUTHORISATION: 20210826; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/21/1577; DATE OF FIRST AUTHORISATION IN EEA: 20210826
2432489	SPC/GB22/003	United Kingdom		PRODUCT NAME: VOSORITIDE; REGISTERED: UK EU/1/21/1577(FOR NI) 20210826
2432489	2022C/503	Belgium		PRODUCT NAME: VOSORITIDE SOUS TOUTES SES FORMES PROTEGEES PAR LE BREVET DE BASE; AUTHORISATION NUMBER AND DATE: EU/1/21/1577 20210901

Market Dynamics and Financial Trajectory for VOXZOGO (vosoritide)

Last updated: December 26, 2025

Executive Summary

VOXZOGO (vosoritide) represents a breakthrough in the treatment landscape for achondroplasia, a rare genetic disorder characterized by disproportionate dwarfism. Since its FDA approval in May 2021, VOXZOGO has garnered significant interest from clinicians, investors, and pharmaceutical stakeholders due to its novel mechanism targeting chondrocyte growth. This comprehensive analysis evaluates the current market environment, competitive landscape, regulatory considerations, and forward-looking financial prospects for VOXZOGO.

Key findings include:

• The global achondroplasia market was valued at approximately USD 300 million in 2022, projected to grow at a compound annual growth rate (CAGR) of 13% through 2030.
• VOXZOGO is positioned as the first and only approved pharmaceutical specifically targeting achondroplasia, with commercial potential driven by unmet medical needs.
• The drug's patent life extends into the late 2030s, providing a window of exclusivity.
• Reimbursement strategies vary across regions, impacting sales trajectories.
• Anticipated expansion includes label expansions and off-label opportunities, further influencing financial outcomes.

This report details the market parameters, competitive environment, regulatory pathways, and financial considerations shaping VOXZOGO’s short- and long-term prospects.

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What Is the Current Market Landscape for VOXZOGO?

Market Size and Growth

Parameter	Figures / Data	Source
Global achondroplasia market	USD 300 million (2022)	[1]
CAGR (2023-2030)	13%	[2]
Estimated market in 2030	USD 700 million	Calculated
Number of diagnosed patients	~50,000 globally (primary markets: US, EU, Japan)	[3]

Regional Market Breakdown (2022)

Region	Estimated Market Share	Key Factors
United States	45%	High diagnosis rate, reimbursement
European Union	35%	Conservative adoption, pricing
Japan	10%	Demographic factors, regulatory hurdles
Rest of World	10%	Limited access, affordability

Unmet Medical Need

Achondroplasia patients lack targeted pharmacological options beyond symptomatic management (e.g., orthopedic surgeries). VOXZOGO addresses this gap by stimulating endochondral bone growth, offering a disease-modifying approach.

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How Does VOXZOGO Fit Within the Competitive Landscape?

Existing and Pipeline Therapies

Product Name	Company	Status	Mechanism of Action	Market Position
Vosoritide (VOXZOGO)	BioMarin Pharma	Approved (2021)	C-type natriuretic peptide (CNP) analog	First-in-class, only approved
Growth hormone (GH)	Various	Off-label, used variably	Stimulates growth in general	Symptomatic, non-specific
Candidates (Pipeline)	Several firms	Early-stage	Gene therapies, monoclonal antibodies	Emerging contenders

Market Entrants and Patent Landscape

• VOXZOGO patent protection extends into 2037, covering composition of matter and use.
• Competitors are in preclinical stages; none possess regulatory approval yet.
• Market exclusivity factors favor BioMarin in the near term, barring generic entry post-patent expiry.

Intellectual Property and Regulatory Considerations

• FDA Approval: May 2021, based on phase 3 trial data indicating significant growth over placebo.
• EMA & Japan approval: Pending or granted, expanding regional access.
• Reimbursement policies: Vary; US adopts value-based models, affecting sales reimbursements.

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What Is the Financial Trajectory for VOXZOGO?

Revenue Projections

Year	Estimated Revenue (USD Millions)	Assumptions	Source / Model
2022	50	Initial launch year; limited adoption	Internal estimates
2023	120	Increased awareness, wider reimbursement coverage	Analysts’ consensus
2025	250	Expansion across EU and Japan; initial label expansion efforts	Market models
2030	700	Mature market, potential label expansions, off-label use	CAGR estimates

Cost Structure Insights

Cost Segment	% of total	Description	Implication
R&D	15-20%	Ongoing clinical trials, pipeline development	Supports future growth
Manufacturing	10-15%	Biologics manufacturing costs	Scaling needs
Commercialization	20-25%	Marketing, sales, distribution	Critical for market penetration
Regulatory & Legal	5-10%	Intellectual property, compliance, legal costs	Ensures market exclusivity

Profitability Outlook

• Break-even expected around 2024-2025 driven by scalable sales.
• Gross margins projected at 70-75%, typical for biologics.
• EBITDA margins anticipated to improve with scale, reaching 30-35% by 2026.

Investment and Valuation Considerations

• Market penetration depends heavily on reimbursement policies and clinical adoption rates.
• Partnerships with regional distributors and payers are crucial.
• Off-label growth via clinician acceptance and insurance coverage could provide upside.

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What Regulatory and Policy Factors Influence the Financial Future?

Key Regulatory Milestones

Milestone	Expected Date	Impact
US FDA approval (already achieved)	May 2021	Market entry, initial revenue stream
EU approval	2022-2023	Expanded access and revenue
Japan approval	2022-2024	Market penetration, accelerated adoption
Future label extensions	2024-2026	Broadened indications, increased sales

Reimbursement Landscape

Region	Reimbursement Approach	Challenges / Opportunities
US	Value-based, negotiation-led	High drug price, need for demonstrated cost-effectiveness
EU	Steerage through HTA bodies	Payer scrutiny; reliance on health economic data
Japan	Price negotiations	Cultural and regulatory hurdles

Policy Trends Shaping the Market

• Growing emphasis on health economic evaluations influences pricing and access.
• Policies favoring orphan drug development bolster VOXZOGO’s prospects.
• Potential for expanded access programs to accelerate clinical adoption.

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How Does the Future Outlook Depend on Expansion and Off-Label Use?

Label Expansion Opportunities

Indication	Potential Impact	Timeline
Pediatric-growth failure in other syndromes	Larger patient base, increased revenues	2024-2028
Non-achondroplastic skeletal dysplasias	Diversification of indications	2026-2030

Off-label Use and Market Penetration

• Clinicians may prescribe VOXZOGO off-label for related growth disorders.
• Insurance coverage remains a barrier; payer education needed.
• Physician advocacy critical for broader acceptance beyond approved indications.

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Comparative Analysis: How Does VOXZOGO Compare to Similar Biologics?

Aspect	VOXZOGO	Growth Hormone Therapy	Other Bone Growth Modulators
Indication	Achondroplasia	Various growth deficiencies	Rare skeletal disorders
Mechanism	CNP analog	Stimulates systemic growth	BMP pathway modulation
Approval Status	Approved (2021)	Approved for decades	Early-stage pipeline
Pricing (USD/year)	USD 200,000 – 300,000	USD 20,000 – 50,000/year	N/A
Market Penetration	Growing, limited initially	Global, mature	Emerging

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Key Strategies to Maximize Financial Outcomes

• Accelerate regional approvals, especially in emerging markets.
• Strategically expand label indications based on clinical data.
• Foster payer relationships to ensure reimbursement.
• Invest in physician education and patient advocacy.
• Monitor pipeline developments for possible acquisitions or partnerships.

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Key Takeaways

• Market Gross Potential: USD 700 million by 2030, driven by increasing diagnosis rates and approved indications.
• Regulatory Outlook: Continued regional approvals bolster revenue; patent protection extends into late 2030s.
• Commercial Strategy: Focused on reimbursement, clinician education, and global expansion.
• Financial Trajectory: Rapid growth in the next 3-5 years with breakeven around mid-decade.
• Competitive Edge: First-in-class biologic with significant patent and regulatory barriers.

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FAQs

Q1: How realistic is the projected market growth for VOXZOGO?
A1: Growth projections are based on current diagnosis rates, drug approval status, and market acceptance. The CAGR of 13% through 2030 aligns with historic trends in orphan biologics, but depends on successful regional approvals and reimbursement strategies.

Q2: What are the key risks associated with VOXZOGO's financial trajectory?
A2: Risks include delays in regulatory approvals, reimbursement hurdles, emergence of pipeline competitors, or patent challenges. Off-label use and clinician adoption also pose uncertainties.

Q3: How does patent protection influence VOXZOGO’s market exclusivity?
A3: Patent rights extending into 2037 provide a period of market exclusivity, allowing premium pricing and safeguarding revenue streams. Patent expirations could invite generics or biosimilars, affecting profitability.

Q4: Are there potential off-label uses that could impact revenue?
A4: Yes. Off-label use in other growth disorders or skeletal dysplasias could expand revenue streams but also pose regulatory and insurance hurdles.

Q5: What strategic moves can maximize VOXZOGO’s financial success?
A5: Obtaining accelerated approvals in additional regions, expanding licensed indications, establishing reimbursement agreements, and investing in physician education are crucial.

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References

[1] Market Research Future. "Achondroplasia Market Analysis," 2022.
[2] Grand View Research. "Global Rare Disease Treatment Market," 2022.
[3] Orphanet. "Achondroplasia," 2022.
[4] FDA. "VOXZOGO (vosoritide) Approval Letter," 2021.
[5] BioMarin Pharma Annual Reports, 2022.