In 2022 , the first results of a 6-month double-blind trial of vamorolone (Agamree®) versus placebo or prednisone were published. This synthetic steroid has been approved in Europe since December 2023 for the treatment of Duchenne muscular dystrophy from the age of 4. New data from this trial, at one year, have just been published.
A total of 121 participants with DMD aged between 4 and 7 years were followed in six cohorts:
– two groups were treated for one year with vamorolone 2 mg/kg/day or 6 mg/kg/day ;
– two groups received prednisone or placebo for 6 months, then were divided into two groups each treated with 2 mg/kg/day or 6 mg/kg/day of vamorolone for the following 5 months. The results showed that :
- vamorolone at 6 mg/kg/day for one year preserves motor skills (6-minute walk, speed of getting up from the ground, climbing 4 steps, etc.), better than at 2 mg/kg/day except for scores measured using the NSAA scale, where the two doses are equivalent, compared with placebo;
- prednisone is more effective than vamorolone (comparison during the first 6 months) but, when patients subsequently take vamorolone, the motor benefits are maintained until the end of the trial;
- prednisone slows growth, which is not the case for vamorolone, and switching to vamorolone after 6 months of prednisone allows a return to a normal growth curve;
- weight gain seems to depend on the patient, but is observed with vamorolone 6 mg/kg/day and prednisone, and is limited with vamorolone 2 mg/kg/day;
- vamorolone remains well tolerated over the long term.
