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27th Aug 2021

After more than 10 years under research and development, 

This is The Day!


BioMarin announced August 27, 2021, that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide) for the treatment of children with achondroplasia starting at age of 2 years until the closure of growth plates, which occurs after puberty. This treatment is administered once a day by injection.

What is VOXZOGO® for achondroplasia and how does it work?

It is the first drug approved for the treatment of children with achondroplasia. It is a modified C-type natriuretic peptide (CNP) that acts at the intracellular level (inside the cells), the chondrocytes, by decreasing the negative signal of fibroblast growth factor receptor 3 (FGFR3) and consequently improving bone growth.

"Today represents an important milestone for the European achondroplasia community. For the first time, medical professionals in Europe can offer a meaningful specific therapeutic treatment option for children and families affected by this condition," said Klaus Mohnike, Professor of Pediatrics and pediatric endocrinologist at Magdeburg University Hospital in Germany and investigator of the Voxzogo clinical program.

Who can receive VOXZOGO®?

It is estimated that more than 11,000 children across Europe, the Middle East and Africa have achondroplasia and may be eligible for treatment with Voxzogo. Approximately one-third of this population is in countries authorized under the European Medicines Agency (EMA) license.

The EMA Committee for Medicinal Products for Human Use (CHMP), adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Voxzogo on the 26th June 2021.

The European Commission has the final decision, which was announced on the 27th Aug 2021, and therefore, Voxgozo, can be marketed in European countries.

And now what comes next for the access of VOXZOGO®?

Now, national approval process comes next. So, from this moment on, the decision to access Voxgozo will be up to each european country, more precisely a decision to be granted by the national agencies or institutes responsible for the evaluation, monitoring and reimbursement of medicines.

The French National Agency for the Safety of Medicines and Health Products (ANSM) has already granted a Temporary Authorization for Use (ATU) to allow immediate access to Voxzogo. This ATU is similar to other Early Access Program (PAP) that exist in other countries, which allow access to drugs not yet approved by the national agencies, when they are indicated for rare conditions with no alternative options, and when the benefit-risk is considered positive.

"We are grateful to the families, researchers and their teams, who have dedicated their time to the clinical program to make this treatment option a reality for this community in the EU. Voxzogo is the most studied therapeutic option for achondroplasia, with an ongoing and robust clinical program built on more than a decade of research and development," said Jean-Jacques Bienaimé, President and CEO of BioMarin.

Safety evaluation of VOXZOGO®

During clinical trials with vosoritide (now voxzogo) approximately 38,000 injections of Voxgozo have been administered to children worldwide to date and Voxzogo was generally well tolerated at all doses. Most adverse effects have been mild and transient and no serious adverse effects have been reported. No adverse effects were found related to worsening of depoporation or bone pathology, and there was no evidence of acceleration of bone age or loss of bone mineral density.

Achondroplasia is a rare, progressive, lifelong condition. It can cause multisystemic complications that, in some cases, require surgical intervention. This approval, proven in a phase 3 clinical trial, is based on improving the speed of growth with increased height, a key determinant in the day-to-day functionality of individuals with this condition. And it is a first step towards understanding the potential benefits of this drug beyond height in the long term.
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