Vosoritide - Generic Drug Details

What are the generic 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 six patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Vosoritide has eighty-three patent family members in twenty-six countries.

One supplier is listed for this compound.

Summary for vosoritide

International Patents:	83
US Patents:	6
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	2
Clinical Trials:	9
Patent Applications:	45
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
Roopa Kanakatti Shankar	Phase 2
UCSF Benioff Children's Hospital Oakland	Phase 1/Phase 2
Andrew Dauber	Phase 2

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	10,646,550		Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-002	Nov 19, 2021		RX	Yes	Yes	8,198,242		Y	Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-001	Nov 19, 2021		RX	Yes	Yes	8,198,242		Y	Y
Biomarin Pharm	VOXZOGO	vosoritide	POWDER;SUBCUTANEOUS	214938-003	Nov 19, 2021		RX	Yes	Yes	11,911,446

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/005475 Voxzogo 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
South Korea	20190119179	씨형 나트륨이뇨 펩티드의 변이체 (VARIANTS OF C-TYPE NATRIURETIC PEPTIDE)
European Patent Office	3328416
Brazil	122019026832	Variantes de peptídeo natriurético tipo c (cnp), seu método de produção recombinante e seu uso, composição farmacêutica, composição para tratamento de uma condição ou distúrbio responsivo a cnp e célula hospedeira para produção in vitro de um peptídeo cnp
China	102481330	Variants of c-type natriuretic peptide

Supplementary Protection Certificates for vosoritide

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
2432489	122022000008	Germany		PRODUCT NAME: VOSORITID IN ALLEN DURCH DAS GRUNDPATENT GESCHUETZTEN FORMEN; REGISTRATION NO/DATE: EU/1/21/1577 20210826
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	22C1004	France		PRODUCT NAME: VOSORITIDE; REGISTRATION NO/DATE: EU/1/21/1577 20210901

Vosoritide Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Vosoritide

Introduction to Vosoritide

Vosoritide, marketed under the brand name VOXZOGO, is a groundbreaking treatment for achondroplasia, the most common form of short-limbed dwarfism. Developed by BioMarin Pharmaceutical Inc., vosoritide has revolutionized the treatment landscape for this genetic disorder.

Mechanism of Action

Vosoritide is a C-type natriuretic peptide (CNP) precursor that targets the underlying genetic causes of achondroplasia. It works by binding to the FGFR3 receptor, thereby inhibiting abnormal bone formation associated with the condition. This unique therapeutic strategy makes vosoritide an attractive and effective treatment option for patients and medical professionals[1].

Market Drivers

Several factors are driving the growth of the vosoritide market:

Increasing Demand and Approval

The U.S. FDA approved vosoritide in November 2021 for improving growth in children with achondroplasia, addressing a significant unmet medical need for over 10,000 children in the United States. This approval has significantly boosted the market demand for vosoritide[4].

Clinical Efficacy and Safety

Real-world evidence studies presented at the European Society for Paediatric Endocrinology (ESPE) meeting in 2024 have further supported the safety and efficacy of vosoritide in children with achondroplasia. These positive results have enhanced the credibility and adoption of vosoritide[3].

Government Funding and Healthcare Infrastructure

Government funding for the treatment of rare genetic disorders, along with advancements in healthcare infrastructure, has driven market growth. Increased awareness and favorable health reimbursement policies, particularly in North America, have also contributed to the market's expansion[1][4].

Financial Trajectory

Revenue Growth

BioMarin has reported significant revenue growth driven by vosoritide. In the third quarter of 2024, revenues from VOXZOGO saw a 54% year-over-year increase, contributing substantially to BioMarin's total revenues of $746 million, a 28% increase from the same period in 2023[2].

Market Size and Projections

The global achondroplasia treatment market, led by vosoritide, is expected to grow from USD 172.96 million in 2023 to USD 816.6 million by 2028, at a CAGR of 36.4%. By 2034, the market is projected to reach USD 3.2 billion, with vosoritide expected to remain a major driver of this growth[1][4].

Geographical Performance

North America, particularly the United States, is a dominant market for vosoritide, driven by favorable health reimbursement policies and increased awareness. The Asia-Pacific region is also expected to show lucrative growth due to its large population, rising healthcare spending, and enhanced healthcare infrastructure[1].

Distribution Channels

Hospital Pharmacies

Hospital pharmacies are anticipated to fuel the market growth due to their specialized expertise, immediate medication availability, and participation in research and clinical trials. This segment's strong connections with healthcare providers and patient networks further support the expansion of vosoritide in the market[1].

Online Pharmacies

The COVID-19 pandemic has accelerated the growth of online pharmacies, which offer convenience and ease of access. This trend is expected to continue, contributing to the overall market growth of vosoritide[1].

Route of Administration

The parenteral route of administration for vosoritide is expected to grow at a healthy CAGR. This is due to its effectiveness in drug delivery, patient compliance, and clinical efficacy, making it a preferred method for administering vosoritide[1].

Impact of COVID-19

The COVID-19 pandemic initially disrupted the achondroplasia treatment market but ultimately boosted demand as healthcare professionals adopted telehealth and remote monitoring technologies. This shift ensured continuous treatment planning for patients with achondroplasia, including those on vosoritide[1].

Challenges and Opportunities

Regulatory Hurdles

Despite the positive market dynamics, regulatory hurdles and the need for long-term follow-up studies remain challenges. However, these challenges also present opportunities for further research and development, potentially leading to new treatment options and market expansion[4].

Psychosocial and Emotional Challenges

Patients with achondroplasia face psychosocial and emotional challenges, which vosoritide can help address by improving their quality of life. This aspect underscores the importance of vosoritide beyond its clinical efficacy, highlighting its potential for long-term market growth[4].

Key Takeaways

• Vosoritide is a groundbreaking treatment for achondroplasia, approved by the FDA in 2021.
• The drug has shown significant clinical efficacy and safety in real-world evidence studies.
• Market growth is driven by increasing demand, government funding, and favorable healthcare infrastructure.
• North America and the Asia-Pacific region are key markets for vosoritide.
• Hospital pharmacies and online pharmacies are crucial distribution channels.
• The parenteral route of administration is preferred due to its effectiveness.

FAQs

Q: What is vosoritide, and how does it work? A: Vosoritide is a C-type natriuretic peptide (CNP) precursor that targets the underlying genetic causes of achondroplasia by binding to the FGFR3 receptor, inhibiting abnormal bone formation.

Q: When was vosoritide approved by the FDA? A: Vosoritide was approved by the U.S. FDA in November 2021 for improving growth in children with achondroplasia.

Q: What are the key market drivers for vosoritide? A: Key market drivers include increasing demand, FDA approval, clinical efficacy, government funding, and favorable healthcare infrastructure.

Q: How has the COVID-19 pandemic impacted the vosoritide market? A: The pandemic initially disrupted the market but ultimately boosted demand as healthcare professionals adopted telehealth and remote monitoring technologies.

Q: What are the projected market sizes for the achondroplasia treatment market? A: The market is expected to grow from USD 172.96 million in 2023 to USD 816.6 million by 2028 and reach USD 3.2 billion by 2034.

Cited Sources

1. Marketdataforecast.com: Achondroplasia Treatment Market Size, Share & Growth, 2032
2. PR Newswire: BioMarin Announces 28% Y/Y Total Revenue Growth in the Third Quarter and Increase in Full-Year 2024 Guidance
3. PR Newswire: BioMarin Presents Real-World Evidence Further Supporting Safety and Efficacy of VOXZOGO (vosoritide) in Children with Achondroplasia
4. GlobeNewswire: Achondroplasia Treatment Market Projected to Reach USD 3.2 Billion by 2034-Unveils Dynamics, Segmentation, and Growth Opportunities[4].