Zolgensma data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing

  • Patients & Caregivers
  • Healthcare Professionals
  • Society & ESG

  • Interim SPR1NT data showed presymptomatic babies with SMA treated with Zolgensma (onasemnogene abeparvovec-xioi) soon after birth achieved age-appropriate motor milestones
  • In addition to meeting both co-primary efficacy endpoints, nine of 22 patients in the completed pivotal STR1VE-US study demonstrated the "ability to thrive," a stringent composite endpoint remarkable compared to untreated children with SMA Type 1
  • Ongoing START long-term follow-up study (cohort 2) demonstrated sustained durability of Zolgensma, including the achievement and maintenance of milestones in the follow up period, now up to 5 years post-dosing and up to 5+ years of age
  • Cumulative safety data from 335 patients treated with Zolgensma indicate a safety profile consistent with previously-reported safety information

Basel, March 24, 2020 AveXis, a Novartis company, today announced a one-time infusion of Zolgensma (onasemnogene abeparvovec-xioi) showed rapid, significant and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated presymptomatically, and sustained durability in patients now up to five years post-dosing and some patients more than five years of age. The STR1VE-US study findings are being presented today during a virtual Clinical Trial Session conducted by the Muscular Dystrophy Association (MDA), scheduled after the 2020 MDA Annual Conference was cancelled due to COVID-19. The accepted posters for SPR1NT, START long-term follow up and cumulative safety data will be published online by MDA in the coming weeks.

Interim data from the ongoing SPR1NT study continue to show patients achieved age-appropriate motor milestones when treated with Zolgensma presymptomatically. Most patients (7/8) with two copies of SMN2 who achieved the ability to sit independently did so within the World Health Organization window of normal development. The six remaining patients in this cohort of 14 patients have not yet passed the developmental window. The importance of independent sitting is that it allows for the potential development and integration of the cognitive, sensory and motor skills that are important for functional independence and social development. Additionally, nearly all patients were fed orally and required no feeding support. Most remained within the age-appropriate weight range. No patients required ventilatory support of any kind.

"SMA is a disease that robs babies of the ability to talk, eat, sit up and even breathe. In complete contrast to the natural course of the disease, patients who received Zolgensma soon after birth before the onset of symptoms are achieving age-appropriate motor milestone development - an extraordinary outcome for SMA patients," said Olga Santiago, M.D., Chief Medical Officer, AveXis. "These SPR1NT data demonstrate the truly transformational impact a one-time dose of gene therapy can have, and further underscore the importance of newborn screening and early intervention to alter the course of the disease."

SMA Type 1 patients experienced rapid, sustained and clinically meaningful improvements in motor function in the completed pivotal STR1VE-US study. In STR1VE-US, nearly all (91%) patients met the co-primary efficacy endpoint of event-free survival at 14 months, and more than half (59%) of patients met the co-primary efficacy endpoint of sitting for