Sarepta’s Exondys 51 Successor On Clinical Hold, But Quick Resolution Expected
The FDA placed a hold on the Phase II study of SRP-5051, which uses the company’s next-generation peptide conjugation technology, but Sarepta thinks it can address safety questions in short order.
You may also be interested in...
The company has initiated the first pivotal double-blind gene therapy trial in DMD in the US in a tight race with Pfizer.
The next-generation DMD drug showed dramatic improvement on exon dystrophin and exon skipping, but small numbers and adverse events give pause.
The RNA drug’s accelerated approval brings Sarepta’s exon-skipping market share to nearly 30%.