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It was back in January 2013, that the pharmaceutical company BioMarin was granted by the European Commission orphan drug designation for Voxzogo, a modified C-type human recombinant natriuretic peptide for the treatment of achondroplasia.

How does voxzogo/vosoritide work?

Bone growth is a dynamic process, for which it is necessary to have a balance between growing and ceasing to grow. During this process, what makes people grow in a controlled way, is the Fibroblast Growth Factor Receptor 3, FGFR3, which has a natural "growth arresting" effect.

In people with achondroplasia, the FGFR3 gene has a mutation, which causes it to become "hyperactive", and as it has the effect of halting growth, it slows down this process considerably.

Voxzogo has an action that partially blocks the hyperactivity of FGFR3, improving the speed of growth.

This drug exists in powder form with solvent to create a daily injectable solution and has now been recommended for marketing from the age of 2 until adolescence. A confirmed diagnosis with genetic testing is required before treatment can be started.

On what is this EMA decision based?

The CHMP opinion is mainly based on a study that evaluated 121 children and young people with achondroplasia (aged between 5 and 18 years). In this study, an average growth improvement of 1.57 cm was observed after one year of treatment. This significant increase occurred proportionally in the spine and lower limbs and the data suggest that this improvement in growth is maintained in the long term.

What are the side effects?

Voxzogo was generally well tolerated at all doses during the clinical trials. Most adverse effects were mild and there is no record of serious effects. There was no evidence of acceleration of bone age or any negative changes in bone density (such as bones becoming more fragile).

The most common side effects observed during the clinical trials were hypotension (low but transient blood pressure), and injection site reactions, also of short duration, among other less frequent ones.

What does this EMA decision mean?

The position taken by the CHMP is an intermediate step on the path of this medicine towards its availability for children and adolescents with achondroplasia. It is still necessary for the Committee on Orphan Medicinal Products (COMP) to reassess whether voxzogo retains the orphan drug designation granted in 2013. This assessment was reviewed by the COMP in June 2021.

The final decision on Market Access Authorisation (MAA) in the EU rests with the European Commission, expected to be granted by September 2021. Once this Market Access Authorisation (MAA) is granted, the technical, therapeutic and price assessment process will be initiated by each Member State.

Key facts:
- Orphan drugs are paid for 100% by the NHS.
- They are not available in pharmacies, but only in hospital pharmacies (inside hospitals) and require a specific prescription.

How much time is still ahead?

The evaluation and approval of orphan drugs is long and may take several months (or even years, depending on each country) between MAA until they can be prescribed by a doctor.

The journey is still long, but the CHMP recommendation is a big step forward in terms of scientific research into treatment for achondroplasia. Current treatment approaches are restricted to supportive care and surgical interventions to increase leg and arm length, correct spinal compression or bowing of the legs.

Sources: Biomarin; EMA

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