Vosoritide - Generic Drug Details

What are the generic drug sources for vosoritide and what is the scope of freedom to operate?

Vosoritide is the generic ingredient in one branded drug marketed by Biomarin Pharm and is included in one NDA. There are seven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

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

One supplier is listed for this compound.

Summary for vosoritide

International Patents:	104
US Patents:	7
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	14
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for vosoritide
What excipients (inactive ingredients) are in vosoritide?	vosoritide excipients list
DailyMed Link:	vosoritide at DailyMed

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

Constraining patent/regulatory exclusivity:

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

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 vosoritide

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

Sponsor	Phase
ICON Clinical Research	PHASE2
BioMarin Pharmaceutical	PHASE2
BioMarin Pharmaceutical	PHASE3

See all vosoritide clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for vosoritide

M05BX	Other drugs affecting bone structure and mineralization
M05B	DRUGS AFFECTING BONE STRUCTURE AND MINERALIZATION
M05	DRUGS FOR TREATMENT OF BONE DISEASES
M	Musculo-skeletal system

US Patents and Regulatory Information for vosoritide

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	214938-002	Nov 19, 2021		RX	Yes	Yes	11,590,204		Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-002	Nov 19, 2021		RX	Yes	Yes	RE48267
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes	11,590,204		Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-001	Nov 19, 2021		RX	Yes	Yes
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes	RE48267
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-001	Nov 19, 2021		RX	Yes	Yes

EU/EMA Drug Approvals for vosoritide

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 vosoritide

Country	Patent Number	Title	Estimated Expiration
European Patent Office	2432489	VARIANTS DU PEPTIDE NATRIURÉTIQUE DE TYPE C (VARIANTS OF C-TYPE NATRIURETIC PEPTIDE)
China	108472336
Canada	2994280
South Korea	20230142644	씨형 나트륨이뇨 펩티드의 변이체 (VARIANTS OF C-TYPE NATRIURETIC PEPTIDE)
China	108472336	C型利尿钠肽变体在治疗骨骼发育不良中的用途 (USE OF C-TYPE NATRIURETIC PEPTIDE VARIANTS TO TREAT SKELETAL DYSPLASIA)
Denmark	3328416

Supplementary Protection Certificates for vosoritide

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2432489	CR 2022 00004	Denmark		PRODUCT NAME: VOSORITIDE; REG. NO/DATE: EU/1/21/1577 20210901
2432489	2290005-4	Sweden		PRODUCT NAME: VOSORITIDE; REG. NO/DATE: EU/1/21/1577 20210921
2432489	LUC00248	Luxembourg		PRODUCT NAME: VOSORITIDE SOUS TOUTES LES FORMES PROTEGEES PAR LE BREVET DE BASE; AUTHORISATION NUMBER AND DATE: EU/1/21/1577 20210901
2432489	SPC/GB22/003	United Kingdom		PRODUCT NAME: VOSORITIDE; REGISTERED: UK EU/1/21/1577(FOR NI) 20210826
2432489	22C1004	France		PRODUCT NAME: VOSORITIDE; REGISTRATION NO/DATE: EU/1/21/1577 20210901
2432489	CA 2022 00004	Denmark		PRODUCT NAME: VOSORITIDE IN ALL FORMS; REG. NO/DATE: EU/1/21/1577 20210901

Market Dynamics and Financial Trajectory for Vosoritide

Last updated: July 27, 2025

Introduction

Vosoritide, a novel pharmaceutical agent developed by BioMarin Pharmaceutical Inc., represents a significant advancement in the therapeutic management of achondroplasia—the most common form of dwarfism. As an injectable C-type natriuretic peptide (CNP) analog, vosoritide targets the underlying genetic pathway involved in cartilage growth, promising to modify disease progression rather than merely managing symptoms. This analysis explores the market landscape, competitive positioning, regulatory developments, and financial prospects shaping vosoritide’s trajectory.

Market Landscape and Therapeutic Need

Achondroplasia affects approximately 1 in 15,000 to 20,000 live births globally, with no approved therapies that alter disease progression until recent developments. Current management strategies focus on symptomatic treatment, including orthopedic interventions and growth hormone therapy, which have limited efficacy and potential side effects. The unmet need for disease-modifying therapies underscores vosoritide’s potential to redefine treatment standards.

The global market for achondroplasia therapeutics is projected to expand as awareness increases and the efficacy and safety profiles of targeted treatments become established. According to industry estimates, the total addressable market could reach hundreds of millions of dollars, factoring in potential repeat dosing, off-label uses, and broader pediatric indications.

Regulatory Milestones and Approvals

Vosoritide's path to market has been marked by significant regulatory progress:

• FDA Approval: In July 2021, the U.S. Food and Drug Administration approved vosoritide for children with achondroplasia aged 5 years and older, marking it as the first targeted therapy for the disorder [1].

• European Commission: Following FDA approval, the European Medicines Agency (EMA) granted marketing authorization in the European Union, broadening access within Europe [2].

• Ongoing Clinical Trials: BioMarin continues Phase 3 trials evaluating long-term safety, efficacy, and expanded age groups, including younger children, which could further expand the indication base.

These milestones establish a robust regulatory foundation, encouraging market penetration and signaling a pathway for future approvals.

Market Entry Strategy and Commercial Dynamics

BioMarin’s approach leverages direct sales, specialized pediatric and genetic clinics, and partnerships with patient advocacy groups. Its emphasis on physician education and patient engagement aims to accelerate adoption. The high cost associated with chronic biologic administration (estimated at $400,000–$500,000 annually per patient) positions vosoritide within premium pharmaceutical pricing dynamics.

Insurance reimbursement, particularly in healthcare systems like the U.S., remains a critical factor. BioMarin is engaging with payers early, emphasizing cost-effectiveness and quality-of-life improvements, which could facilitate favorable reimbursement terms.

Competitive Landscape

Currently, vosoritide is the pioneering drug targeting the underlying molecular pathway in achondroplasia; no direct competitors have advanced similarly. However, several potential contenders are in preclinical or early clinical stages, including:

• Setruschedin: A gene therapy approach aiming to induce endogenous CNP production.
• Other CNP analogs or modulators: Under development by smaller biotech firms.

The rarity of achondroplasia limits the number of direct competitors, but the overall therapeutic pipeline’s maturity remains modest, granting vosoritide a first-mover advantage. Nevertheless, competition from symptomatic treatments and gene therapy modalities could influence market share dynamics.

Financial Trajectory and Revenue Projections

Market Penetration and Adoption

BioMarin projects initial sales to be incremental, targeting highly specialized centers. Early adoption is driven by the unmet need and regulatory endorsement. Over a 5- to 10-year horizon, increasing prescription volume is anticipated with expanded approvals, demonstrated long-term efficacy, and wider age group coverage.

Pricing and Revenue Assumptions

Assuming a price point of approximately $400,000–$500,000 annually per patient, revenue projections hinge on:

• Patient Population: Estimated to reach 3,000–5,000 patients globally, accounting for diagnosed, eligible, and treated individuals.
• Market Penetration: Initially capturing 10–20% of the potential market within 3–5 years, increasing as indications expand.

Long-term Financial Outlook

With sustained demand, a well-established reimbursement pathway, and minimized competition, vosoritide's revenue could grow substantially. BioMarin's 2022 revenue from orphan drugs exceeded $2 billion, with significant contribution from other rare disease therapies, providing a financial backbone to support vosoritide’s commercialization.

Projected revenues may range from $200 million in early years to over $1 billion annually within a decade, contingent on approval expansion, market uptake, and pricing strategies. Cost considerations include R&D, manufacturing scale-up, marketing, and payer negotiations.

Market Risks and Opportunities

Risks

• Regulatory Delays: Additional trials or safety concerns could hinder approval or limit indications.
• Reimbursement Challenges: High treatment costs necessitate favorable payer acceptance; unfavorable coverage decisions could restrict access.
• Competitive Threats: Emergence of alternative therapies or gene editing treatments could dilute market share.

Opportunities

• Expanded Indications: Pediatric populations below age 5 and adults with achondroplasia approximate a multi-hundred million dollar market.
• Global Expansion: Access in emerging markets broadens revenue streams.
• Combination Therapies: Potential to enhance efficacy by combining vosoritide with other agents.

Conclusion

Vosoritide’s market dynamics are shaped by its groundbreaking mechanism, regulatory milestones, and high unmet need. While initial sales are modest relative to blockbuster drugs, the long-term financial trajectory is promising, driven by expanding indications, increasing physician familiarity, and favorable pricing. Successful navigation of reimbursement landscapes and competition management will be key to maximizing revenue potential.

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Key Takeaways

• Vosoritide sets a new standard as the first disease-modifying therapy for achondroplasia, with significant upside potential.
• Regulatory approvals in the U.S. and EU have laid a strong foundation for commercial success.
• High treatment costs necessitate strategic payer engagement to ensure broad access.
• The total addressable market could reach several billion dollars over the next decade, contingent on expanded indications.
• Competition remains limited currently but warrants monitoring as the specialty evolves.

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FAQs

1. What is the current status of vosoritide’s regulatory approval?
Vosoritide received FDA approval in the U.S. in July 2021 for children aged 5 and above with achondroplasia and has secured approval from the EMA for European markets, signaling robust regulatory acceptance.

2. How does vosoritide differ from existing treatments for achondroplasia?
Unlike symptom-focused therapies, vosoritide targets the genetic pathway responsible for abnormal cartilage growth, offering potential disease modification and growth improvement.

3. What is the expected timeline for market expansion and broader adoption?
Post-approval, early adoption is anticipated within high-volume specialized centers. Expanding age indications and accumulating long-term efficacy data could accelerate adoption over 3–5 years.

4. What are the main economic considerations for investors assessing vosoritide’s market potential?
High pricing, limited current competition, a sizable unmet need, and expanding indications support strong revenue growth, though reimbursement hurdles and potential competition pose risks.

5. Are there any ongoing clinical trials that could impact vosoritide’s market position?
Yes, BioMarin is conducting trials in younger children and long-term studies, which may enable broader indications and enhance market penetration if successful.

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References:

[1] U.S. Food and Drug Administration. (2021). FDA Approves Vosoritide for Achondroplasia.
[2] European Medicines Agency. (2021). Marketing Authorization for Vosoritide in the EU.