AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)

After a manufacturing-related delay, Novartis has won conditional approval in the EU for the one-time gene therapy to treat patients with a clinical diagnosis of SMA type 1 and others with up to three copies of the SMN2 backup gene, the company said on May 19th. Zolgensma has demonstrated significant and clinically meaningful therapeutic benefit in pre-symptomatic and symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease and to date, sustained for 5 years post-dosing. Immediate access to Zolgensma, aligned to the label, is available in France through the ATU framework and expected shortly in Germany
AveXis in discussions with EU governments and reimbursement agencies to agree on

For further information, see Novartis (https://www.novartis.com/news/media-releases/avexis-receives-ec-approval-and-activates-%22day-one%22-access-program-zolgensma-only-gene-therapy-spinal-muscular-atrophy-sma)

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