Voxzogo

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

Voxzogo was more effective than placebo at increasing growth rate in a second study involving 75 children aged from 4 months to less than 5 years with confirmed achondroplasia. The main measure of effectiveness was change in height Z-score, a measure which compares the patient’s height to the average expected for their age and gender. After one year of treatment, children given Voxzogo had on average a greater improvement in height Z-score than those given placebo. Children who received Voxzogo also grew about 0.8 cm more during the year of treatment than those who received placebo.

For the full list of side effects and restrictions with Voxzogo, see the package leaflet.

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.

Voxzogo is effective at increasing the growth rate in children with achondroplasia aged 4 months and older. This may increase final height, allowing persons with achondroplasia to perform daily activities more easily. The side effects of the medicine are considered manageable. The safety profile in children below 5 years of age is similar to that in older children. The European Medicines Agency decided that Voxzogo’s benefits are greater than its risks and it can be authorised for use in the EU.

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.

The medicine can only be obtained with a prescription. Treatment with Voxzogo must be started and supervised by a doctor experienced in the treatment of growth disorders or abnormal growth of bones.

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.

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.