BRIEF—Zolgensma picks up Japan approval in SMA

Novartis has picked up Japanese approval for its one-time gene therapy Zolgensma (onasemnogene abeparvovec), for spinal muscular atrophy (SMA) in young children.

The therapy is backed by clinical data showing prolonged event-free survival and achievement of motor milestones, marking new territory in the treatment of the disease.

Untreated, SMA leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.

The firm said it expects to secure reimbursement from the Japanese authorities by the middle of the year. The therapy will be available immediately.