Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA)1
* SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 22,3* Zolgensma (onasemnogene abeparvovec) is approved for the treatment of pediatric SMA patients with 3 or fewer copies of the SMN2 gene, or infantile-onset SMA1* Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene to halt disease progression1
DORVAL, QC, Dec. 16, 2020 /CNW/ – Novartis Pharmaceuticals Canada Inc. is pleased to announce that Health Canada has approved Zolgensma® (onasemnogene abeparvovec) an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 3 or fewer copies of SMN2 gene; or infantile-onset SMA1.
Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defective SMN1 gene1. It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1gene into a patient’s cells, halting disease progression and restoring production of SMN protein1.
“SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking. Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease,” said Dr. Hugh McMillan, Pediatric Neurologist at the Children’s Hospital of Eastern Ontario in Ottawa. “The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy. The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy.”
“When I first started diagnosing SMA, I couldn’t have imagined that we would see such scientific advancements,” said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mère Enfant Soleil, Université Laval in Québec City. “Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones.”
In Canada each year, approximately one in 10,000 babies are born with SMA, a rare, genetic neuromuscular disease caused by a defective or missing SMN1 gene3. Without a functional SMN1 gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or...