Novartis has released new Zolgensma (onasemnogene abeparvovec-xioi) data, demonstrating rapid, significant and clinically meaningful therapeutic benefit of the drug in patients with spinal muscular atrophy (SMA).
The proprietary gene therapy was analysed 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.
Additionally, interim data from the ongoing SPR1NT study continue to show patients achieved age-appropriate motor milestones when treated with Zolgensma presymptomatically.
“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, 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.”
Novartis confirmed that all patients in this study who received the therapeutic dose were alive and free of permanent ventilation and continued to maintain developmental milestones, including two patients who achieved the new milestone of standing with assistance during the long-term follow-up period.
Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion.
SMA is the leading genetic cause of infant death. If left untreated, SMA type I leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.