VOXZOGO (vosoritide) - Achondroplasia in patients 2 years to under 5 years of age

The Committee deems that the clinical benefit of VOXZOGO (vosoritide) powder and solvent for solution for subcutaneous injection is low within the scope of the MA indication covered by this assessment:

• Patients from 4 months to under 2 years of age (MA indication extension);
• Patients from 2 years to under 5 years of age (the new data supplied in the context of the reassessment are of a nature to modify the Committee’s previous conclusions).

Considering:

• the results of a randomised, double-blind, placebo-controlled phase 2 study having evaluated vosoritide in patients from 4 months to under 5 year of age, which do not enable a robust conclusion to be reached concerning any superiority compared to placebo after 52 weeks of treatment for the main growth endpoints assessed, in particular the clinically relevant primary endpoint, which was variation in height Z-score (difference not statistically significant), both in the cohort of patients from 4 months to under 2 years of age and in the cohort of patients from 2 years to under 5 years of age,
• the level of evidence of these new data in patients from 2 years to under 5 years, which is superior to that of the data available for the initial assessment, which were purely descriptive and concerned 4 patients,
• the absence of a demonstrated benefit on quality of life in this population, despite this being particularly impacted in this condition,
• the major methodological limitations of the external comparisons supplied, which do not enable any formal conclusion to be reached with respect to a potential longer-term benefit of vosoritide versus no treatment,
• the safety profile, globally similar to that already observed in patients 5 years of age and older, but marked by more injection site reactions, particularly in the youngest patients,

the Committee deems that VOXZOGO (vosoritide) powder and solvent for solution forsubcutaneous injection provides no clinical added value (CAV V) compared with the usual management of achondroplasia within the scope of the MA indication covered by this assessment:

• Patients from 4 months to under 2 years of age (indication extension);

Patients from 2 years to under 5 years of age (the new data supplied in the context of the reassessment are of a nature to modify the Committee’s previous conclusions).