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
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?
Find answers to common questions
Talk to your child about treatment
Helpful information about Voxzogo
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.
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
IN THE NEWS
21 SEPTEMBER 2023 – BioMarin to Present Data Showing Long-Term Benefit of VOXZOGO® (vosoritide) on Growth in Children with Achondroplasia at 2023 European Society for Paediatric Endocrinology (ESPE) Meeting
14 MARCH 2023 – BioMarin to Share Updated Data at 2023 American College of Medical Genetics and Genomics (ACMG) Meeting Demonstrating Commitment to Understanding Long-Term Benefit of VOXZOGO® in Children with Achondroplasia
21 JUNE 2022 – BioMarin Announces the Ministry of Health, Labor and Welfare (MHLW) in Japan Granted Approval for VOXZOGO® (vosoritide) for Injection for the Treatment of Children with Achondroplasia, Whose Growth Plates are Not Closed
13 JUNE 2022 – BioMarin Announces Favorable Results from Global Phase 2 Study of VOXZOGO™ (vosoritide) for Injection in Infants and Young Children with Achondroplasia at the Endocrine Society Annual Meeting, ENDO 2022 (June 11-14), in Atlanta
15 APRIL 2021 – BioMarin Announces New and Updated Data at 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting Demonstrating Commitment to Understanding Achondroplasia and Potential Treatment Choice
20 MARCH 2021 – BioMarin Announces Oral Presentation at ENDO2021, the Endocrine Society’s Annual Meeting, with Data Demonstrating 2 Years of Treatment Benefit in Children with Achondroplasia Treated with Vosoritide
9 NOVEMBER 2020 –BioMarin Expands Vosoritide Clinical Program
8 SEPTEMBER 2020 – BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia
20 APRIL 2016 – BioMarin Provides Program Update on Vosoritide in Achondroplasia
12 OCTOBER 2015 – BioMarin’s Initial 6-Month Data from Phase 2 Study of Vosorotide (BMN 111) in Children with Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting
17 SEPTEMBER 2013 – BioMarin Provides Highlights Ahead of Research and Development Day
26 SEPTEMBER 2012 – BioMarin Announces Phase 1 Results for BMN-111 for Achondroplasia
3 JANUARY 2012 – BioMarin Announces IND for BMN-111 for Achondroplasia is Active
8 DECEMBER 2011 – Highlights from BioMarin’s Research & Development Day