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Novartis: AveXis Gets EC Approval For Zolgensma; Starts "Day One" Access Program

Swiss drug major Novartis (NVS) announced Tuesday that its affiliated gene therapy company AveXis has received the European Commission's conditional approval for Zolgensma (onasemnogene abeparvovec) to treat spinal muscular atrophy or SMA. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

With the EC approval, Zolgensma can be used to treat patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene.

The company noted that 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.

The company noted that immediate access to Zolgensma, aligned to the label, is available in France through the ATU framework and expected shortly in Germany.

Further, AveXis has activated "Day One" access program for Zolgensma, the only gene therapy for SMA. The program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments.

In Europe, SMA's cumulative estimated healthcare costs per child ranges between 2.5 million euros to 4 million euros within the first 10 years alone.

AveXis is in talks with EU governments and reimbursement agencies to agree on terms of innovative "Day One" access program to enable rapid access in all EU countries given urgent need to treat SMA.

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