Novartis: AveXis says trial of gene therapy was positive

(CercleFinance.com) - AveXis, a Novartis unit, said on Thursday that its gene therapy Zolgensma showed significant therapeutic benefit in children with spinal muscular atrophy (SMA).


Interim data from Phase 3 trials, including pre-symptomatic patients, showed positive outcomes, demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival in patients with SMA Type 1, the group said.

It is often considered critical to diagnose SMA and begin treatment as early as possible in order to stop irreversible motor-neurone loss and therefore enable crawling, sitting and walking.

The data will be presented during the 2019 European Paediatric Neurology Society (EPNS) congress.

Zolgensma has been recently apporved by the FDA, but AveXis has subsequently informed health regulators about "data manipulation," which impacts the accuracy of certain data regarding the product.

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