Some disappointing news for the Duchenne community in the U.S. yesterday. Sarepta’s drug golodirsen for patients amenable to exon 53 received a complete response letter from the FDA based on 2 safety concerns. What this means is the FDA has competed their review of this new drug submission and decided not to approve if for marketing in its present form. Sarepta plans to work with the agency to address the issues and find a pathway forward.
To date we are unaware of any submission to Health Canada and this will mean more delays to access for those boys affected with this mutation.
Let’s hope they find a quick solution.