VIMIZIM (elosulfase alfa) - Mucopolysaccharidosis type IVA (Morquio A Syndrome)

The Committee deems that the clinical benefit of VIMIZIM (elosulfase alfa) remains substantial in the MA indication.

Considering:

• the initial data assessed by the Committee, which had demonstrated an efficacy of VIMIZIM (elosulfase alfa) only in the 6 Minute Walk Test (MWT) in studies conducted over the short term (48 weeks), with no demonstrated benefit in terms of the clinical signs of the disease and the long-term prognosis,
• new clinical data with a longer follow-up (120 weeks), but which nonetheless remains limited given the slow progression of this disease,
• the limitations of these data that concern only patients not severely affected by the disease since capable of walking, the absence of information concerning the impact of VIMIZIM
  (elosulfase alfa) on the evolution of height, a clinically relevant endpoint in this disease,
• analysis of data from the registry, which does not eliminate the uncertainties concerning the efficacy of VIMIZIM (elosulfase alfa) raised in the clinical studies,
• the lack of information on the impact of treatment with VIMIZIM (elosulfase alfa) on a potential reduction in the need for surgery, which can be a source of sometimes fatal complications,
• the absence of analysable results on the impact of VIMIZIM (elosulfase alfa) treatment on quality of life in this disease that has a significant impact on this,
• an efficacy of VIMIZIM (elosulfase alfa), which appears to be modest overall,
• its safety profile, which appears to be favourable overall despite the potential onset of serious infusion reactions, but taking into account:

• the seriousness of the disease,
• the partially met medical need,

the Committee deems that VIMIZIM (elosulfase alfa) provides a minor clinical added value (CAV IV) in the current care pathway which includes only symptomatic supportive care, with no other medicinal products being authorised.