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Phase III SPR1NT and RESTORE study data on Zolgensma shows efficacy and safety in spinal muscular atrophy.- Novartis

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Published:16th Mar 2021
Novartis announced new data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA). The overall safety profile remains favorable following presymptomatic treatment, in the long-term follow-up period from clinical studies and in the real-world setting. New data underscore the critical importance of identifying and treating SMA as early as possible. In contrast to the natural history of this devastating disease, which leads to progressive and irreversible loss of motor function, children treated with Zolgensma presymptomatically in the Phase III SPR1NT trial achieved age-appropriate motor milestones within the World Health Organization (WHO) window of normal development – including sitting, standing and walking – were able to eat exclusively by mouth and did not require ventilatory support of any kind. There were no serious, treatment-related adverse events reported in SPR1NT. In addition, children identified via newborn screening enrolled in the RESTORE real-world registry were significantly less likely to receive more than one SMA therapy compared with those who were diagnosed clinically. Long-term follow-up data from two studies continued to demonstrate that children treated with Zolgensma experienced a sustained benefit from gene therapy in the years following dosing, with no evidence of new or delayed safety signals. Zolgensma led to achievement of new milestones years after treatment – including sitting – with sustained durability in children now up to six years old and more than five years post-treatment. Emerging findings from the RESTORE registry – designed to provide real-world data for enhancing our understanding of patients cared for in routine practice – indicate older children (at least 6 months) achieved a clinically meaningful benefit when treated with Zolgensma alone, after switching to gene therapy or in combination with another SMA therapy, with safety events consistent with the previously described safety profile. Nearly all children with two or more CHOP INTEND assessments available improved or maintained their scores, and most had a clinically meaningful minimum 4-point increase. These data were presented during the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference.
Condition: Spinal Muscular Atrophy
Type: drug

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