ROG/SRPT: Duchenne EMBARK likely enought to lessen age restrictions

Despite the headline failure of the phase 3 EMBARK trial in Duchenne, the FDA accepted an efficacy supplement aimed to remove the 4-5y age restriction and the ambulatory requirement and to convert to a traditional approval, based on data from both the new EMBARK and existing ENDEAVOR trials. We assess the likelihood of the FDA converting Elevidys’s approval from accelerated to traditional, lessening the current 4-5y age restriction and the likelihood of the removal of the ambulatory restriction