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
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.
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
IN THE NEWS
20 OCTOBER 2023 – U.S. Food and Drug Administration Approves BioMarin’s VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia
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
15 SEPTEMBER 2023 – BioMarin Receives Positive CHMP Opinion in Europe to Expand Use of VOXZOGO® (vosoritide) to Treat Children Aged 4 Months and Older with Achondroplasia
27 FEBRUARY 2023 – BioMarin Announces Record Fourth Quarter and Full Year 2022 Total Revenues Driven by Strong Global Demand for VOXZOGO® and Steady Growth of Enzyme Business
27 AUGUST 2021 – European Commission Approves BioMarin’s VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
3 MARCH 2021 – BioMarin Completes Full Enrollment in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
21 DECEMBER 2020 – BioMarin Announces Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study
9 NOVEMBER 2020 –BioMarin Expands Vosoritide Clinical Program
2 NOVEMBER 2020 – Food and Drug Administration Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia
11 SEPTEMBER 2020 – BioMarin Announces Presentation of Vosoritide Phase 3 Data in Children with Achondroplasia at the American Society for Bone and Mineral Research 2020 Annual Meeting
20 AUGUST 2020 – BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia
13 AUGUST 2020 – European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Vosoritide to Treat Children with Achondroplasia
23 JULY 2020 – BioMarin Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia
16 DECEMBER 2019 – BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide
14 JUNE 2018 – BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia
12 DECEMBER 2016 – BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia
19 OCTOBER 2016 – BioMarin Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics Meeting
20 APRIL 2016 – BioMarin Provides Program Update on Vosoritide in Achondroplasia
18 APRIL 2016 – BioMarin to Highlight Breath of Innovative Development Pipeline at R&D Day on April 20th in New York
17 JUNE 2015 – BMN-111 (vosoritide) Improves Growth Velocity in Children with Achondroplasia in Phase 2 Study
14 JANUARY 2014 – BioMarin Doses First Patient in Phase 2 Trial With BMN-111 for Treatment of Children With Achondroplasia
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
16 FEBRUARY 2012 – BioMarin Initiates Phase 1 Trial for BMN-111 for the Treatment of Achondroplasia
3 JANUARY 2012 – BioMarin Announces IND for BMN-111 for Achondroplasia is Active
8 DECEMBER 2011 – Highlights from BioMarin’s Research & Development Day
19 OCTOBER 2010 – BioMarin Announces Program for BMN-111 for the Treatment of Achondroplasia