Novartis reports interim long-term follow-up data from Phase 1 START trial
AveXis, a Novartis company, announced interim long-term follow-up data from the Phase 1 START trial of the investigational product Zolgensma that showed durability of the gene therapy in patients with spinal muscular atrophy, or SMA, Type 1 nearly four years after treatment. These data were presented during the 2019 American Academy of Neurology Annual Meeting, the company noted. As of March 8, 2019, all patients maintained the motor function and milestones gained during the trial following treatment with Zolgensma. In addition, no patients had any additional requirements for ventilatory or nutritional support. Two of the four patients who required Bilevel Positive Airway Pressure support at the beginning of the long-term follow-up study no longer required it regularly. No new treatment-related adverse events have emerged during the follow-up period. Patients in START were treated with gene therapy alone during the 24-month study duration. In the long-term follow-up study, 7 of 10 patients remain on monotherapy alone. Initiation of combination therapy was at parental and physician discretion and was not due to loss of motor function, the company said.