Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
The latest data from two LTFU studies LT-001 and LT-002 was presented at the 2023 MDA Clinical & Scientific Conference.
Interim results from the 15-year LT-002 study was also presented. All patients (100 percent) were shown to maintain motor milestones achieved during their respective parent studies in the follow-up period.
Results from the IV-administered cohort, which included 63 patients, demonstrated a single administration of Zolgensma provided consistent, substantial and durable efficacy over time.
On 16 March 2023, the National Institute for Health and Care Excellence (NICE) recommended Zolgensma as an option in babies with presymptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.
Imran Kausar, General Manager at Novartis Gene Therapies UK commented on NICE’s decision: “Zolgensma will be the first treatment to be routinely commissioned for presymptomatic babies in England, and as it is imperative to diagnose SMA and begin treatment as early as possible, we welcome the decision by NICE for this recommendation.”
There is no national newborn screening programme for SMA in the UK. Infants without a family history are only diagnosed if symptoms are identified. As this can take up to six months, an established programme is important.
Following decision by the NICE committee that onasemnogene abeparvovec is effective in treating presymptomatic SMA, final guidance is expected to be published on 19 April 2023.
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