Voxzogo

Voxzogo is given as an injection under the skin once a day, preferably around the same time of day. The site of injection should be changed with each injection. The recommended dose is calculated according to the patient’s body weight. The usual daily dose is 15 microgram per kg body weight.

Treatment should only be started when achondroplasia is confirmed by genetic testing and should end when the patient is not likely to grow any further.

Voxzogo injections can be given by the patients’ caregiver once they have been trained appropriately.

For more information about using Voxzogo, see the package leaflet or contact your doctor or pharmacist.

In patients with achondroplasia, the FGFR3 gene, which regulates growth, is permanently 'switched on'. This prevents normal growth of bones, leading to bones that are shorter than normal. The active substance in Voxzogo, vosoritide, works by binding to a receptor (target) called natriuretic peptide receptor type B (NPR-B), which reduces the activity of FGFR3. This stimulates growth of bones, thereby improving the symptoms of the disease. 

Voxzogo was more effective than placebo (dummy treatment) at increasing growth rate after 52 weeks of treatment in a study involving 121 children (aged 5-17 years) with confirmed achondroplasia. Children who received Voxzogo grew about 1.57 cm more during the one year of treatment than those receiving placebo. In addition, the results suggest that the improvement in growth is maintained.

Voxzogo is also expected to work well in younger patients (aged 2-5 years) as the underlying disease and the way Voxzogo works are the same across the age groups.

The most common side effects with Voxzogo (which may affect more than 1 in 10 people) are injection site reactions (such as swelling, redness, itching or pain), vomiting and decreased blood pressure. For the full list of side effects and restrictions, see the package leaflet.

The main study has shown that Voxzogo is effective at increasing growth rates in children with achondroplasia, which could result in a substantial improvement in final height if confirmed and allow persons with achondroplasia to perform daily activities more easily. The side effects are considered manageable. The European Medicines Agency decided that Voxzogo’s benefits are greater than its risks and it can be authorised for use in the EU.

The company that markets Voxzogo will provide the additional data on the effectiveness and safety of the medicine from an ongoing study involving younger patients (aged 2-5 years).

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Voxzogo have been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Voxzogo are continuously monitored. Suspected side effects reported with Voxzogo are carefully evaluated and any necessary action taken to protect patients.

Voxzogo received a marketing authorisation valid throughout the EU on 26 August 2021.