Eteplirsen has received support from medical experts and parents of affected Duchenne muscular dystrophy children, but the FDA has delayed their decision for approval of this drug. Here, the authors present the results of the first pooled-analysis of previous studies assessing the safety and efficacy of eteplirsen. A total of four relevant clinical studies were identified. A pooled-analysis was performed using data relating to percentage dystrophin-positive fibres obtained from muscle biopsy, and the six-minute walk test (6 MWT). The average increase in percentage dystrophin-positive fibres after treatment with eteplirsen was 24.23% (range -4 to 78; SD 24.44%). The average rate of decline in distance walked was 65metres (range -335 to 83; SD 100.08 m). Whether or not this increase in percentage dystrophin-positive fibres and distance walked is clinically significant is unclear, and there is therefore a need for more clinical trials.
