Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients who have three or fewer copies of the SMN2 gene, or with infantile-onset disease. “Today’s announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine…

Most children with spinal muscular atrophy (SMA) show an abnormal increase in the levels of liver enzymes — suggestive of liver damage — following treatment with Zolgensma, according to a study by Novartis, the therapy’s developer. This known side effect of the gene therapy was mainly very mild and…

Infants with spinal muscular atrophy (SMA)  type 1 who received a single infusion of Zolgensma continue to achieve developmental milestones and show improvements in motor function, according to interim data from the Phase 3 STR1VE-EU trial.  Importantly, these improvements were also seen in infants with more severe forms of…

Novartis will open a new clinical trial investigating Zolgensma, given via an injection directly into the spinal canal, after the U.S. Food and Drug Administration (FDA) recommended a “pivotal confirmatory study” into the gene therapy’s use in older spinal muscular atrophy (SMA) patients. Findings from this future…

England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy (SMA), according to a press release from SMA UK. NICE had previously planned to conduct evaluations of clinical trial and cost-effectiveness data, with…

Novartis’ gene therapy Zolgensma is generally safe and effective in promoting motor improvements in children with spinal muscular atrophy (SMA) treated up to age 2, according to a multicenter study. However, “if the children are older and heavier, there is a potential for more liver injury [a known side effect…

The U.S. Food and Drug Administration (FDA) has given Catalent Biologics approval to aid in manufacturing products for use with AveXis’ spinal muscular atrophy (SMA) gene therapy, Zolgensma, at its Maryland facility. The FDA inspected the company’s commercial-scale gene therapy manufacturing center in June. Catalent and AveXis,…

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which included children treated with Zolgensma at older ages than those reported in clinical trials, suggested…

Biogen has announced plans to launch a Phase 4 clinical trial evaluating the benefits of Spinraza (nusinersen) in infants and children with spinal muscular atrophy (SMA) who were previously treated with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi). The company expects to enroll the first patients in…

Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional SMA types, according to a consensus statement from a panel of European experts. The statement covers 11 points, and highlights that clinical trials of Zolgensma to date involved a…