AGAMREE (vamorolone) - Duchenne muscular dystrophy (DMD)

The Committee deems that the clinical benefit of AGAMREE 40 mg/mL (vamorolone) oral suspension is moderate in the MA indication.

Considering:

• the non-analysable results for the comparison between vamorolone and prednisone, scheduled in the hierarchical sequence of secondary endpoints in the comparative study versus placebo and versus prednisone,
• the results relative to the superiority of vamorolone versus placebo for the primary endpoint, i.e. the change from baseline in Time to Stand Test (TTSTAND) velocity in patients treated with vamorolone 6 mg/kg versus placebo at week 24,
• the lack of analysable results in this study for quality of life criteria, since these were the subject of a descriptive evaluation only,
• the safety profile of vamorolone in the study versus placebo and versus prednisone, which does not appear to be more favourable than that of prednisone; in particular, in the same way as with glucocorticoid therapy, weight gain was observed during treatment with vamorolone,
• uncertainties with respect to the long-term safety of vamorolone; in particular, analysis over a limited time of 30 months does not make it possible to confirm the expected positive effect on children’s growth, and there are no data available on a potential effect of vamorolone on puberty,

the Committee deems that AGAMREE 40 mg/mL (vamorolone) oral suspension provides no clinical added value (CAV V) compared to the prednisone-based drugs used off-label, but recommended in the treatment of Duchenne muscular dystrophy.