19 March 2020 - Reimbursement with MHLW is expected by the end of 1H20, pending agreement, Zolgensma will be available at that time.
Novartis Pharma today announced that the Japanese Ministry of Health, Labour and Welfare approved Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) in patients under the age of two, including those who are pre-symptomatic at diagnosis. Patients must be negative for elevated anti-AAV9 antibodies.
A rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, SMA results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.