New treatment for people with dwarfism

News 25/06/2021

EMA has recommended granting a marketing authorisation in the European Union (EU) for Voxzogo (vosoritide) for the treatment of achondroplasia, a condition that impairs bone growth and causes dwarfism. The medicine is intended for use in patients 2 years and older whose epiphyses (growth plates of the bones) are not yet closed. The condition must be confirmed by genetic testing before patients can start treatment with Voxzogo.

People with achondroplasia typically are of short stature. Some may also experience serious health complications, such as compression of the spinal cord, bowed legs, ear infections or heart problems, which can increase the risk of people dying prematurely.

Options for those affected by the condition are limited to supportive care and surgical interventions to extend limb length, correct spinal compression or bowed legs. Most patients are diagnosed in early infancy or at birth. It is estimated that 350 children are born with achondroplasia every year in the EU.

In patients with achondroplasia the fibroblast growth-factor receptor 3 (FGFR3) gene is permanently ‘switched on’, preventing normal bone growth. Vosoritide, the active substance in Voxzogo, works by attaching to a receptor called natriuretic peptide receptor type B (NPR-B) on the surface of cells. The activation of this receptor will reduce the activity of FGFR3 and thereby stimulate the normal growth of bones and improve the symptoms of the disease.

Voxzogo is available as powder and solvent for solution for injection.

The opinion of EMA’s human medicines committee (CHMP) is mainly based on one study which evaluated 121 patients with achondroplasia who were between 5 and 18 years of age. During the course of the study, a statistically significant improvement in growth was observed in patients treated with vosoritide compared to placebo, adding an average 1.57 cm in height after a year of treatment. The observed increase in growth occurred proportionally in both the spine and the lower limbs. Longer-term data in number of patients suggest that the improved growth is maintained. A separate ongoing study including subjects aged 2 to 5 also suggests consistent improvement in growth.

The most common side effects observed in clinical trials were hypotension (low blood pressure), injection site reactions and vomiting.    

The opinion adopted by the CHMP is an intermediary step on Voxzogo’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.


  1. The application for Voxzogo is BioMarin International Limited from Ireland.
  2. Voxzogo was designated as an orphan medicinal product on 24 January 2013.
  3. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.

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