Novartis announced that Zolgensma (onasemnogene abeparvovec) had received conditional approval in Europe for treating patients with spinal muscular atrophy (SMA). The drug is indicated in patients with a clinical diagnosis of SMA Type 1 or SMA patients having three copies of the SMN2 gene. The approval is for pediatric patients whose body weight is less than 21kg.
The company announced that the drug is available for immediate access in France through the ATU framework and expected to be available in Germany shortly.
The approval is based on Phase STR1VE-US and Phase 1 START trials, which were intended to evaluate the efficacy and safety of Zolgensma in pediatric patients. The drug demonstrated prolonged event-free survival and rapid motor function improvement. Patients were able to sit without support, crawl and walk.
“SMA Europe receives with deep excitement the news on the approval by the European Commission, of a gene therapy for treating a part of our community,” said Mencía de Lemus, President of SMA Europe.
As per “Day One” access program
Novartis will provide rebates in order to align the cost of early access with negotiated prices
RESTORE is a registry of patients diagnosed with SMA across world and reimbursement bodies will get the access
About Spinal Muscular Atrophy
Spinal Muscular Atrophy is a rare genetic neuromuscular disorder that leads to permanent ventilation in more than 90% of the cases in infants. This is caused by a lack of functional SMN1 gene, which leads to a gradual loss of motor neurons, impacting the functioning of muscles.
Zolgensma acts by providing a functional copy of the human SMN gene. The drug has already been approved in the US and Japan. The company is anticipating the approvals in Switzerland, Canada, Australia, Argentina, South Korea, and Brazil.