Sarepta discontinues next generation of exon-skipping drugs for Duchenne muscular dystrophy
Sarepta announced that it is discontinuing its next generation of exon-skipping drugs for Duchenne muscular dystrophy over safety concerns. In addition to its much-debated gene therapy and other approved exon-skipping drugs for particular Duchenne mutations, Sarepta had been developing follow-on treatments designed to be more targeted toward muscle cells.
Seven severe treatment-related adverse events arose in a recent clinical trial for the successor, though, and while Sarepta previously argued the data were still positive, executives said that the FDA told them they would not be able to file for accelerated approval. Sarepta opted to scrap all its experimental drugs that use the same chemistry after doing its own risk-benefit calculation, Sarepta CEO Doug Ingram said. The setback could leave room for other competitors with targeted exon skippers to beat out Sarepta in a lucrative market, but trials are still early and they have also faced some safety concerns. “I wish these folks well,” Ingram told investors. “I think they have a high bar and a long road to get to the right place.”
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