Novartis’s Zolgensma Receives EC approval for Spinal Muscular Atrophy

The European Commission has granted conditional approval for Zolgensma for treatment of two forms of chromosome 5q-related spinal muscular atrophy (SMA) in babies and young children up to 21 kilograms.  

The approval was based on the results of two clinical trials in which Zolgensma (onasemnogene abeparvovec-xioi) demonstrated prolonged event-free survival and rapid motor function improvement in patients less than six months in age.

The company is in talks with EU governments and reimbursement agencies to expand access to the drug.