New study shows benefit of Novartis' SMA gene therapy

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Novartis says updated data has confirmed the significant clinical benefit of its gene therapy ZOLGENSMA (onasemnogene abeparvovec) for patients with spinal muscular atrophy.

The company, which gained rights to the one-time treatment ZOLGENSMA through its acquisition of AveXis, has presented study findings that show the prolonged benefit of the gene therapy, including in patients treated pre-symptomatically, up to five years post-dosing and some patients more than five years of age.

According to the company, the findings continue to show patients achieved age-appropriate motor milestones when treated with ZOLGENSMA pre-symptomatically, including the ability to sit independently, while nearly all patients were fed orally and required no feeding support. 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 Dr Olga Santiago, Chief Medical Officer of Novartis' 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.”

The company said data showed a range of other clinically meaningful improvements in motor function, including with the inclusion of a composite endpoint covering functions of swallowing, feeding and age-appropriate weight maintenance.

The federal government's Medical Services Advisory Committee could consider ZOLGENSMA for funding during 2020.