Sarepta’s Next-Gen DMD Drug SRP-5051 Shows Advantages, But Safety Concerns Too
The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
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The company plans to start its Phase III trial of SRP-9001 in the middle of this year, with a potential readout by the end of 2022.
Arising from an Oxford and Cambridge university collaboration, the spin-out has selected Duchenne muscular dystrophy as the first target for its peptide platform.
Sarepta cited two safety issues when announcing a complete response letter for its exon 53 skipping DMD drug golodirsen, but the US FDA's decision may be a result of the Exondys 51 approval from 2016, analysts suggest.