BioMarin Unveils Promising Growth Data for VOXZOGO Therapy

BioMarin Pharmaceutical Inc. Highlights VOXZOGO Efficacy

BioMarin Pharmaceutical Inc. recently announced significant data from the ongoing CANOPY clinical program evaluating VOXZOGO (vosoritide). This program is particularly focused on children with achondroplasia and other related genetic skeletal conditions. The results, which will be presented at an upcoming international meeting, showcase impressive findings that demonstrate how VOXZOGO not only promotes growth but also improves health-related quality of life (HRQoL) in the affected children.

Enhancements in Health-Related Quality of Life and Physical Functioning

The data from BioMarin indicate remarkable improvements in several areas of health-related quality of life for children undergoing treatment with VOXZOGO. Caregivers have reported that the therapy has positively influenced physical performance, emotional well-being, and social interactions among children with achondroplasia. These improvements have included enhanced motor skills — such as reaching, walking, and cycling — which families have recognized as vital for their children's development.

Bone Growth Without Compromising Strength

Recent analyses from a Phase 2 study shed light on another critical aspect of VOXZOGO: its ability to promote bone lengthening while maintaining the integrity of bone strength. This is significant for children with achondroplasia, as stronger bones can help prevent potential fractures and complications often associated with the condition.

Encouraging Data in Related Genetic Conditions

Investigators are also exploring the effects of VOXZOGO in children with other conditions linked to genetic skeletal abnormalities, including hypochondroplasia and Noonan syndrome. The collected data suggest a sustained growth benefit and favorable safety profile across these studies. Early results have shown that participants in these trials experienced beneficial outcomes similar to those seen in achondroplasia.

First Approved Treatment Bridging Critical Gaps

Serving as the first approved treatment for children with achondroplasia, VOXZOGO represents a groundbreaking advance in the management of this genetic condition. As stated by Dr. Hank Fuchs, president of Worldwide Research and Development at BioMarin, the continued investigation into VOXZOGO highlights the urgency and necessity in addressing unmet needs within this patient population.

Continuous Commitment to Research and Development

The ongoing CANOPY clinical program continues to evaluate the safety and efficacy of VOXZOGO, exploring additional applications in multiple genetic skeletal disorders. The vision behind this expansive research is to broaden treatment options for children facing the challenges that these conditions present.

The Global Reach and Acceptance of VOXZOGO

Currently, VOXZOGO has been approved in the United States, Japan, Australia, and the EU to facilitate linear growth in children diagnosed with achondroplasia. This widespread acceptance underscores the therapy's promising clinical results and the critical role it plays in supporting the growth and development of pediatric patients.

About BioMarin

Founded in 1997, BioMarin is dedicated to advancing treatments for rare genetic disorders. With a strong focus on leveraging genetic discoveries, the company has developed transformative therapies aimed at improving the quality of life for patients with significant medical needs.

Frequently Asked Questions

What is VOXZOGO?

VOXZOGO (vosoritide) is a prescription medication used to promote growth in children with achondroplasia, acting on the underlying genetic factors that limit bone growth.

How does VOXZOGO improve health-related quality of life?

Caregiver reports indicate that VOXZOGO positively impacts physical abilities, emotional well-being, and social engagements, significantly enhancing the quality of life for children treated with it.

What conditions beyond achondroplasia is VOXZOGO being studied for?

Research is being conducted on the efficacy of VOXZOGO for conditions such as hypochondroplasia and Noonan syndrome, among others.

Where is VOXZOGO available?

VOXZOGO is currently approved for use in the United States, Japan, Australia, and within the European Union, providing a worldwide treatment option for eligible children.

What is BioMarin's mission?

BioMarin aims to transform the lives of patients with rare genetic disorders by developing therapies that address the root causes of these conditions, leveraging innovative research and development.

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