Vosoritide for Dwarfism

Recruiting at 22 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: BioMarin Pharmaceutical

Must not be taking: Growth hormone, IGF-1, Anabolic steroids

Disqualifiers: Short stature, Unstable condition, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it does exclude participants who are taking certain prohibited medications or have been treated with growth hormone, IGF-1, or anabolic steroids.

How is the drug vosoritide unique for treating dwarfism?

Vosoritide is unique because it is the first approved drug specifically for achondroplasia, a common cause of dwarfism, and works by targeting the genetic mutation that affects bone growth. It is administered as a daily injection at home, which is different from other treatments that may not directly address the underlying genetic issue. ^{1 2 3 4 5}

What data supports the effectiveness of the drug Vosoritide for treating dwarfism?

Vosoritide has been shown to increase growth velocity in children with achondroplasia, a common cause of dwarfism, and has received approval from both the US FDA and the European Medicines Agency. Clinical studies have demonstrated its ability to help children grow by targeting the genetic cause of achondroplasia. ^{1 2 3 4 6}

Who Is on the Research Team?

Medical Director, MD

Principal Investigator

BioMarin Pharmaceutical

Are You a Good Fit for This Trial?

This trial is for children with hypochondroplasia, a form of short-limbed dwarfism. Participants should have conditions like dwarfism, lordosis (curved lower back), or congenital limb deformities related to HCH.

Inclusion Criteria

I am a female over 10 or have started my periods and can take pregnancy tests.

If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study

I am either male or female.

Exclusion Criteria

Have known hypersensitivity to vosoritide or its excipients

Short stature condition other than HCH

I might need surgery for an unstable condition during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily subcutaneous injections of vosoritide or placebo for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Vosoritide

Trial Overview The study is testing Vosoritide's effectiveness compared to a placebo in treating children with HCH. It's a Phase 3 trial, which means it's looking at how well the drug works and monitoring its safety.

How Is the Trial Designed?

Group I: vosoritide injection with vial and syringe Experimental Treatment 1 Intervention

Group II: Placebo injection with vial and syringe Placebo Group 1 Intervention

Vosoritide is already approved in United States for the following indications:

Approved in United States as Voxzogo for:

• To increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses
• to increase linear growth in pediatric patients less than 5 years of age with achondroplasia with open epiphyses

Who Is Running the Clinical Trial?

BioMarin Pharmaceutical

Lead Sponsor

Trials

162

Recruited

115,000+

Alexander Hardy

BioMarin Pharmaceutical

Chief Executive Officer since 2023

MBA from INSEAD

Greg Friberg

BioMarin Pharmaceutical

Chief Medical Officer

MD from New York Medical College

Published Research Related to This Trial

Vosoritide (Voxzogo) has received accelerated approval from the FDA for treating children aged five and older with achondroplasia, a common form of dwarfism.

To ensure safety and prevent hypotensive episodes, children taking vosoritide should consume a meal and 240 to 300 mL of fluid within an hour before administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Drug Treats Pediatric Achondroplasia. Aschenbrenner, DS.[2023]

Vosoritide, administered at a recommended dose of 15 μg/kg, was shown to be effective in increasing growth velocity in children with achondroplasia over a 52-week phase III study involving 60 patients, with no significant safety concerns noted.

The pharmacokinetics of vosoritide indicated rapid absorption and consistent exposure without accumulation, and the development of anti-vosoritide antibodies in 42% of patients did not adversely affect growth outcomes or drug exposure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Chan, ML., Qi, Y., Larimore, K., et al.[2022]

Voxzogo (vosoritide) is the first drug approved by the FDA and EMA specifically for treating achondroplasia, a leading cause of dwarfism, marking a significant advancement in addressing this rare condition.

Despite the risk of decreased blood pressure as a severe adverse event, the benefits of Voxzogo in fulfilling an unmet medical need for patients with achondroplasia led to its accelerated approval in the U.S. as a treatment for a rare pediatric disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update. Simran, ., S, KDS., Dushantrao, SC., et al.[2023]

Vosoritide is the first approved therapy specifically designed to increase growth velocity in children with achondroplasia, and early experiences from experts indicate a generally positive reception in clinical practice.

Effective management of vosoritide treatment requires multidisciplinary support, patient education on administration, and monitoring for adherence, as these factors significantly influence treatment outcomes and patient satisfaction.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice. Semler, O., Cormier-Daire, V., Lausch, E., et al.[2023]

Vosoritide (VOXZOGO®) is a modified C-type natriuretic peptide that effectively treats achondroplasia by inhibiting the overactive FGFR3 gene, which negatively regulates bone growth.

Approved in the EU for patients aged 2 years and older with open epiphyses, vosoritide represents a significant advancement in achondroplasia treatment, with ongoing regulatory reviews in the USA and further clinical development worldwide.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide: First Approval. Duggan, S.[2022]

Vosoritide is the first approved treatment for achondroplasia, and parents of children using it reported a strong motivation to manage the daily injectable treatment for potential long-term benefits, such as improved height and independence.

The study highlighted the need for better support and consistent training for families, as experiences with treatment initiation varied significantly, impacting their ability to manage the treatment effectively at home.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia. NiMhurchadha, S., Butler, K., Argent, R., et al.[2023]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

New Drug Treats Pediatric Achondroplasia. [2023]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. [2022]

3. Japan pubmed.ncbi.nlm.nih.gov

Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Vosoritide: First Approval. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia. [2023]