VOXZOGO (vosoritide)

Opinions on drugs - Posted on Feb 04 2022

Reason for request

First assessment

Key points

Favourable opinion for reimbursement in the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.

What therapeutic improvement?

Therapeutic improvement in the current management of patients with achondroplasia.

Role in the care pathway?

Role of the medicinal product in the care pathway

The proprietary medicinal product VOXZOGO (vosoritide), as a daily subcutaneous injection, is the first treatment with an MA in achondroplasia.

It is a first-line treatment in patients 2 years of age and older with achondroplasia whose epiphyses are not closed. It should be initiated as early as possible. According to the SPC, this medicinal product should be discontinued once the patient has stopped growing.

VOXZOGO (vosoritide) should be initiated and directed by a physician appropriately qualified in the management of growth disorders or skeletal dysplasias.

Clinical Benefit

Substantial

The Committee deems that the clinical benefit of VOXZOGO (vosoritide) is substantial in the MA indication.

Clinical Added Value

moderate

Considering:

demonstration of the superiority of vosoritide compared to placebo in terms of annual height increase, a clinically relevant primary endpoint, with an effect size that is also clinically relevant (+1.57 cm/year) in a randomised double-blind study in children over 5 years of age,
the unmet medical need in this rare and disabling genetic condition, for which management to date has been limited to symptomatic treatment,

And despite:

data available in only four children between the ages of 2 and 5 years, although there is a benefit in treating this condition as early as possible,
the absence of robust data relative to quality of life, which is particularly impacted in this condition,
uncertainties with respect to the long-term safety profile of VOXZOGO (vosoritide) and the maintenance of its efficacy beyond the age of 2 years, in the absence of data,
the absence of available data on the impact of this treatment on the other clinical manifestations of the condition,

The Committee considers that VOXZOGO (vosoritide) provides a moderate clinical added value (CAV III), in the care pathway compared to the current management, in the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed.