AveXis, the Novartis-held biotechnology company based in the U.S., has reportedly declared data from the ongoing trials of Zolgensma (onasemnogene abeparvovec-xioi, AVXS-101) that exhibited positive results in a broad selection of patients afflicted with SMA (Spinal Muscular Atrophy).
Authentic sources close to the matter stated that the data includes the first presentation from the Phase 1 STRONG trial that exhibited motor function gains & milestone achievements through IT (intrathecal) delivery in patients with SMA Type 2; new data from Phase 3 STR1VE trial, which showed increase in motor functions and prolonged event-free survival, and first data presentation from the Phase 3 SPR1NT trial, that showed motor milestone achievement coupled with normal development in SMA patients who are treated pre-symptomatically. Sources further added that these data have been presented at the AAN (American Academy of Neurology) 2019 Annual Meeting.
SMA, for those uninitiated, is a critical neuromuscular disorder characterized by the loss of motor neurons resulting in progressive muscle weakness & paralysis. It generally occurs due to a defect in the SMN1 gene which codes SMN – a significant protein for survival of motor neurons.
According to David Lennon, President, AveXis, the company has witnessed Zolgensma offering speedy motor function improvement, prolonged survival and milestone achievements with a single, one-time dose. He went on to add that the robust data presented at the AAN signifies a growing body of evidence supporting Zolgensma’s usage as a potential, foundational therapy for the SMA treatment across a huge population.
Zolgensma is an investigational gene therapy which is in development at present as a one-time infusion for the SMA Type1. It was developed in close collaboration with Genethon, and in December 2018, the Food & Drug Administration (FDA) approved the Biologics License Application of the company for use of Zolgensma in in SMA Type 1 patients.
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