CLINICAL TRIALS PROFILE FOR VOSORITIDE

All Clinical Trials for Vosoritide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02055157 ↗	A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia	Completed	BioMarin Pharmaceutical	Phase 2	2014-01-13	This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.
NCT02724228 ↗	A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)	Active, not recruiting	BioMarin Pharmaceutical	Phase 2	2016-01-26	This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
NCT03197766 ↗	A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia	Completed	BioMarin Pharmaceutical	Phase 3	2016-12-12	The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
NCT03424018 ↗	An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia	Active, not recruiting	BioMarin Pharmaceutical	Phase 3	2017-12-12	The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
NCT03583697 ↗	A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia	Active, not recruiting	BioMarin Pharmaceutical	Phase 2	2018-05-23	Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia.
NCT03989947 ↗	An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia	Enrolling by invitation	BioMarin Pharmaceutical	Phase 2	2019-06-12	This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.
NCT04219007 ↗	Vosoritide for Selected Genetic Causes of Short Stature	Recruiting	Andrew Dauber	Phase 2	2020-08-04	Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte. The study team hypothesizes that patients with selected genetic causes of short stature that interact with this pathway will benefit from treatment with vosoritide, a CNP analog, a selective NPR-B agonist which directly targets the growth plate. This study will enroll patients with short stature in selected genetic categories and will follow them for a 6 month observation period to obtain a baseline growth velocity, safety profile and quality of life assessment. Patients will then be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes.

Clinical Trial Conditions for Vosoritide

Condition Name

Condition Name for Vosoritide
Intervention	Trials
Achondroplasia	6
Short Stature	2
Turner Syndrome	1
MPS IVA	1

Condition MeSH

Condition MeSH for Vosoritide
Intervention	Trials
Achondroplasia	6
Dwarfism	2
Gonadal Dysgenesis	1
Mucopolysaccharidosis IV	1

Clinical Trial Locations for Vosoritide

Trials by Country

Trials by Country for Vosoritide
Location	Trials
United States	49
Australia	10
United Kingdom	6
Japan	4
Germany	2

Trials by US State

Trials by US State for Vosoritide
Location	Trials
California	7
Texas	6
Illinois	6
Wisconsin	4
Ohio	4

Clinical Trial Progress for Vosoritide

Clinical Trial Phase

Clinical Trial Phase for Vosoritide
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	6
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for Vosoritide
Clinical Trial Phase	Trials
Active, not recruiting	3
Not yet recruiting	2
Completed	2

Clinical Trial Sponsors for Vosoritide

Sponsor Name

Sponsor Name for Vosoritide
Sponsor	Trials
BioMarin Pharmaceutical	6
Andrew Dauber	1
UCSF Benioff Children's Hospital Oakland	1

Sponsor Type

Sponsor Type for Vosoritide
Sponsor	Trials
Industry	6
Other	3

Vosoritide: Clinical Trials, Market Analysis, and Projections

Introduction to Vosoritide

Vosoritide, marketed as VOXZOGO, is a recombinant C-type natriuretic peptide analogue developed by BioMarin Pharmaceutical. It is designed to increase annualized growth velocity in children with certain genetic growth disorders, most notably achondroplasia, the most common form of short-limbed dwarfism.

Clinical Trials Overview

Achondroplasia Trials

In a recent Phase 2 trial, vosoritide was evaluated in children with achondroplasia aged 3-59 months. The study, conducted across 16 hospitals in Australia, Japan, the UK, and the USA, involved 75 participants who received either vosoritide or a placebo for 52 weeks. The results showed a mild adverse event profile and a significant gain in the change in height Z score from baseline, with a least-squares mean difference of 0.25 (95% CI -0.02 to 0.53) between the vosoritide and placebo groups[1].

Hypochondroplasia Trials

Vosoritide has also been tested in children with hypochondroplasia, another genetic cause of short stature. The first global Phase II study for vosoritide in this population demonstrated an increased average growth rate of 1.8 cm annually. The study involved 24 participants and showed an increase in the height standard deviation score (SDS) by 0.36 SD during the 12-month treatment period. The annualized growth velocity increased by 2.26 standard deviations, and the hypochondroplasia-specific height SDS increased by 0.38 SD[2][4].

Other Growth-Related Conditions

In addition to achondroplasia and hypochondroplasia, vosoritide is being investigated for its efficacy in other growth-related conditions, including idiopathic short stature (ISS) and Noonan syndrome. Data presented at the 2024 Pediatric Endocrine Society (PES) Annual Meeting indicated positive results for these conditions, suggesting that vosoritide could be a precision therapy for multiple genetic disorders affecting growth[3].

Safety and Tolerability

The clinical trials have generally shown that vosoritide is well-tolerated. In the trial involving children with achondroplasia aged 3-59 months, adverse events were common but mostly transient and related to injection-site reactions. Serious adverse events were reported in both the vosoritide and placebo groups, but the overall safety profile was favorable[1].

For children with hypochondroplasia, the medication was also well-tolerated, with some patients showing excellent responses to the treatment. Dr. Andrew Dauber, Chief of Endocrinology at Children’s National Hospital, noted that the medication's tolerability and efficacy support further research in this area[2][4].

Market Analysis

Current Market Position

Vosoritide, under the brand name VOXZOGO, was approved by the FDA in November 2021 and has since generated significant revenue. In 2023, it generated $470 million in revenue, leveraging its first-to-market advantage[5].

Competition

A key competitor to vosoritide is TransCon CNP, developed by Ascendis Pharma. Following positive results from its Phase III ApproaCH Trial, TransCon CNP is gaining ground with analysts revising their revenue forecasts upward. While TransCon CNP is expected to generate $26 million in revenue in 2024, its dosing advantage of weekly injections compared to vosoritide's daily injections could be a significant factor if it is approved by the FDA and EMA in 2025[5].

Market Projections

Despite the emergence of TransCon CNP, vosoritide is expected to maintain a strong market position due to its established presence and ongoing clinical success. However, the market share and revenue projections for vosoritide may be adjusted downward as TransCon CNP moves closer to approval. The probability of FDA approval for TransCon CNP has risen to 84.5%, which could impact vosoritide's future market dominance[5].

Patient Benefit and Quality of Life

The clinical trials have shown that vosoritide can significantly improve the quality of life for children with genetic growth disorders. For example, Ivan Maric’s 11-year-old daughter, Mia, who has hypochondroplasia, experienced life-changing improvements, including increased independence in everyday tasks such as washing her hair and reaching the sink to wash her hands[4].

Future Research and Development

Vosoritide's mechanism of action, which targets the ERK1/2-MAPK pathway, suggests potential applications in various genetic conditions affecting growth. Ongoing and future trials will further explore its efficacy in conditions like ISS, Noonan syndrome, NPR2 mutations, and Aggrecan mutations. The positive results from current trials provide a proof of principle that vosoritide could be a versatile treatment option for multiple growth-related disorders[3][4].

Key Takeaways

• Clinical Efficacy: Vosoritide has demonstrated significant growth benefits in children with achondroplasia and hypochondroplasia.
• Safety Profile: The medication is generally well-tolerated with a mild adverse event profile.
• Market Position: Vosoritide retains a strong market position but faces emerging competition from TransCon CNP.
• Patient Benefit: Vosoritide improves the quality of life for children with genetic growth disorders.
• Future Research: Ongoing trials are exploring vosoritide's potential in other growth-related conditions.

FAQs

What is vosoritide and how does it work?

Vosoritide is a recombinant C-type natriuretic peptide analogue that increases annualized growth velocity by targeting the ERK1/2-MAPK pathway, leading to increased chondrocyte proliferation and differentiation.

Which conditions is vosoritide approved for?

Vosoritide is currently approved for increasing linear growth and opening growth plates in children with achondroplasia.

What are the common side effects of vosoritide?

Common side effects include transient injection-site reactions and injection-site erythema, with serious adverse events being relatively rare.

How does vosoritide compare to its competitor, TransCon CNP?

Vosoritide has a first-to-market advantage but faces competition from TransCon CNP, which offers a dosing advantage of weekly injections compared to vosoritide's daily injections.

What is the current market revenue for vosoritide?

In 2023, vosoritide generated $470 million in revenue.

Are there ongoing trials for vosoritide in other conditions?

Yes, vosoritide is being investigated for its efficacy in conditions such as idiopathic short stature (ISS), Noonan syndrome, NPR2 mutations, and Aggrecan mutations.

Sources

1. Vosoritide therapy in children with achondroplasia aged 3-59 months. PubMed.
2. Novel medicine could address short stature in children. European Pharmaceutical Review.
3. New Data for BioMarin's VOXZOGO® (vosoritide) for Multiple Growth Related Conditions in Children. BioMarin Pharmaceutical.
4. First global clinical trial achieves promising results for hypochondroplasia. Children’s National Hospital.
5. TransCon CNP Gains Ground Against Voxzogo with Positive Results and Higher Approval Odds. Visible Alpha.

Last updated: 2025-01-01