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.3 Approximately 60% of all SMA is Type 1. Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene. Zolgensma is administered during a single intravenous (IV) infusion, delivering a new working copy of the SMN gene into a patient's cells, halting disease progression. Approximately 15-20 SMA patients in
'SMA is the leading genetic cause of infant death and, if left untreated in its most common form, Type 1, leads to death or the need for permanent ventilation by the age of two in more than 90% of cases,' said Kazunari Tsunaba, president and representative director, Novartis Pharma. 'A one-time dose of Zolgensma has the potential to make a truly transformative impact on this life-threatening disease. This is an important day for the children and families in
Approval is based on the Phase 1 START, START Long-term follow-up, Phase 3 STR1VE-US, Phase 3 SPR1NT and Phase 1/2 STRONG (intrathecal injection) trials. START and STR1VE-US were designed to evaluate the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients
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