Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy has failed to meet its primary objective in a phase 3 trial, but the company says it will file to extend the use of the medicine regardless.
The EMBARK study of Elevidys (delandistrogene moxeparvovec) was designed to support the expansion of the label for the recently approved gene therapy to include all ambulatory DMD patients, rather than its current age range of four to five.
