Roche says Elevidys benefit-risk in ambulatory patients remains positive

Roche (RHHBY) in its earnings release provided safety update on Elevidys gene therapy for Duchenne muscular dystrophy in non-ambulatory patients. The company partners with Sarepta (SRPT) outside the U.S. Roche said, “After a thorough clinical review, the benefit-risk profile of Elevidys in non-ambulatory patients with Duchenne has been re-assessed following two cases of fatal acute liver failure. Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting. Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified. The benefit-risk profile of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged.”