FDA Approves Controversial Gene Therapy for All Duchenne Muscular Dystrophy Patients

Decision overruled internal experts despite failed clinical trials, raising concerns about regulatory standards.

Elevidys gene therapy now approved for all ages with Duchenne muscular dystrophy, regardless of mobility status.
FDA's Peter Marks overruled objections from three expert review teams and two directors to expand approval.
Critics argue the decision undermines scientific rigor and could erode trust in the FDA.
The therapy, costing $3.2 million per patient, missed its primary trial goals but showed some secondary benefits.
Families of patients advocate for the treatment, citing urgent need despite mixed clinical results.