BIOMARIN

BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.¹ On October 19, 2010, BioMarin announced their program for BMN-111 for the treatment of achondroplasia. 11 years later, on November 19, 2021, BioMarin received FDA approval for VOXZOGO™ (vosoritide) for injection, indicated to increase linear growth in children with achondroplasia aged 5 and up with open growth plates. On October 20, 2023, the FDA approved VOXZOGO® for children under 5 years with achondroplasia.

VOXZOGO IS THE FIRST FDA-APPROVED PHARMACEUTICAL TREATMENT FOR ACHONDROPLASIA

get more in the know about achondroplasia

What is Voxzogo?

VOXZOGO® (vosoritide) is an FDA-approved treatment that helps increase linear growth in children with achondroplasia aged 5 years and up whose growth plates are still open.²

HOW DOES VOXZOGO WORK?

VOXZOGO is a molecule that stimulates the CNP pathway. It works alongside the body’s natural CNP to stimulate the CNP signaling pathway to promote bone growth. See more here.

how can I get voxzogo?

Discuss VOXZOGO® (vosoritide) with your child’s doctor. Click here to find a physician in your area who prescribes VOXZOGO.
Once you receive a prescription and are enrolled in BioMarin RareConnections^TM, a Clinical Coordinator will contact you to discuss your insurance coverage and financial options.
Your BioMarin Clinical Coordinator will coordinate with a specialty pharmacy to ship a supply of VOXZOGO to your home.
A healthcare provider will train you how to give your child VOXZOGO before you use it at home for the first time. Do not start administering injections until you’ve completed this training.
BioMarin Clinical Coordinators are available for virtual and at-home injection training reinforcement and follow-up education.

vosoritide for hypochondroplasia and other genetic conditions

On November 9, 2020, BioMarin announced they were expanding their clinical program for vosoritide with two new Phase 2 studies. The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression. The second study is an investigator-initiated study sponsored by Children’s National Hospital in Washington, D.C., to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000. One of the genetic conditions included in the investigational study is hypochondroplasia. This study is led by Dr. Andrew Dauber, Chief of Endocrinology at Children’s National Hospital.

UNDERSTANDING HYPOCHONDROPLASIA
Hypochondroplasia (HCH) is related to achondroplasia but typically has more subtle features that are not noticed right at birth. HCH is caused by a mutation in the fibroblast growth factor receptor-3 (FGFR3) gene. Typically, the FGFR-3 protein functions in a signaling pathway to slow linear growth in the growth plates of the long bones. The gene change that causes hypochondroplasia turns on the pathway more than it should, albeit to a lesser degree than achondroplasia, so bones grow slower and end up shorter than they typically would. This type of change associated with an increased ability is called a “gain-of-function” mutation.³