With 48-week data from a Phase II open-label trial in Duchenne muscular dystrophy (DMD) showing an advancement in clinical benefit beyond what a 24-week readout showed last year, Wave Life Sciences said on 26 March that it is on track to file its exon 53-skipping oligonucleotide candidate WVE-N531 for accelerated approval in the US in 2026.
Wave Aspires To Accelerated Approval In DMD With 48-Week Data
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
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