ent ventilation or death by age 2 in more than 90% of cases.SMA is the leading cause of genetic infant death.[4] Approximately 450 to 500 infants are born with SMA in the US annually.
It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.This is especially critical in the most severe form where degeneration starts shortly before birth and escalates quickly.
With states adding SMA to their genetic newborn screening panel, babies with SMA can begin to be widely identified at birth and the ability to have earlier intervention can be improved.
"Zolgensma's one-time dose of gene therapy has the potential to make a truly transformative impact on this life-threatening disease," said Kenneth Hobby, president of Cure SMA, a patient advocacy organization dedicated to the care, treatment and cure of SMA. "Our organization is leading the way to a world without SMA and we are excited the FDA's approval of Zolgensma brings patients and families a powerful new treatment which corrects the underlying cause of the disease."
The approval of Zolgensma is based on data from the ongoing Phase 3 STR1VE trial and the completed Phase 1 START trial eva luating the efficacy and safety of a one-time IV infusion of Zolgensma in patients with SMA Type 1 who showed symptoms of SMA at <6 months of age, with one or two copies in the STR1VE trial or two copies in the START trial of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations.These data show Zolgensma provides unprecedented rates of survival never seen in the natural history of the disease; rapid motor function improvement, often within one month of dosing; and, durable milestone achievement, including the ability to sit without support, a milestone never achieved in untreated patients.
Safety observations in STR1VE were comparable to those seen in the START trial. The most commonly observed adverse events were elevated aminotransferases and vomiting.
"We are grateful to the tenacious researchers, partners and families who participated in the Zolgensma clinical trials that helped us achieve this incredible milestone," said Dave Lennon, president of AveXis. "We are proud to bring this one-time gene therapy to pediatric patients with SMA and remain committed to advancing the science behind Zolgensma to transform SMA, as well as other rare genetic diseases."
About Zolgensma Clinical Data
The efficacy of Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was eva luated in STR1VE, an open-label, single-arm clinical trial (ongoing), and in START, an open-label, single-arm, ascending-dose clinical trial (completed). Patients experienced onset of clinical symptoms consistent with SMA before 6 months of age. All patients had genetically confirmed bi-allelic SMN1 gene deletions, two copies of the SMN2 gene, and absence of the c.859G>C modification in exon 7 of SMN2 gene (which predicts a milder phenotype). All patients had baseline anti-AAV9 antibody titers of >= 1:50, measured by ELISA. In both trials, Zolgensma was delivered as a single-dose intravenous infusion.
Efficacy was established on the basis of survival, and achievement of developmental motor milestones such as sitting without support. Survival was defined as time from birth to either death or permanent ventilation. Permanent v |
