The Phase III STR1VE data show prolonged event-free survival, early and rapid increases in The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) and significant milestone achievement in SMA Type I.

AveXis, a Novartis company since 2018, announced that 21 patients were alive and event-free as of September 27 last year, with an event defined as either death or at least 16 hours a day of breathing ventilation support for 14 consecutive days. The median age at the time of analysis was 9.5 months, "with six of seven patients who could have reached 10.5 months of age or older surviving event-free."

The data "reinforce what was seen in the pivotal Phase 1 START trial, including trends toward prolonged survival and milestone achievement never seen in the natural history of the untreated disease," said Olga Santiago, MD, Chief Medical Officer, AveXis.

She continued, "With a patient population and baseline characteristics closely matched to the START trial, these data build upon the body of evidence supporting the use of Zolgensma for SMA Type I."

The trial included 22 patients who carry at least one copy of the SMN2 backup gene and who have bi-allelic SMN1 gene deletion or point mutations, and who are less than six months of age at the time of gene therapy.

Spinal muscular atrophy (SMA) is a genetic condition that makes the muscles weaker and causes problems with movement, including floppy or weak arms and legs, twitching or shaking muscles and sometimes breathing difficulties.

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