EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EURoche believes the benefit-risk remains positive in the ambulatory Duchenne populationElevidys is the first and only disease-modifying gene therapy for DMD Basel,

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could further jeopardize Sarepta’s financial future.

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.