Sarepta has announced the discontinuation of its next-generation exon-skipping drugs aimed at treating Duchenne muscular dystrophy, citing significant safety concerns observed in recent clinical trials. This decision follows discussions with the FDA, which indicated that accelerated approval for these drugs would not be feasible given the safety profile.
The experimental drugs, designed to be more targeted towards muscle cells, are based on the same chemical platform. During trials, seven severe treatment-related adverse events were reported, prompting a comprehensive risk-benefit assessment by Sarepta. CEO Doug Ingram stated that the company opted to halt development across its pipeline of drugs utilizing this chemistry.
Sarepta continues to market its existing approved exon-skipping therapies and gene therapy for Duchenne muscular dystrophy. The discontinuation of the next-generation drugs, however, creates an opportunity for competitors developing targeted exon skippers. Ingram acknowledged the challenges ahead for these companies, stating, "I wish these folks well. I think they have a high bar and a long road to get to the right place."
The move comes as a setback for Sarepta's Duchenne program, but the company is focusing on its existing portfolio and monitoring the progress of competitors in the field. The Duchenne muscular dystrophy treatment landscape remains competitive, with multiple companies pursuing novel therapeutic approaches.
