AveXis receives EC approval and activates ?Day One? access program for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA)
- Zolgensma??(onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene
- 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 terms of innovative ?Day One? access program to enable rapid access in all EU countries given urgent need to treat SMA
- Retroactive rebates ensuring early access costs are aligned with negotiated prices following local clinical and economic assessment processes
- Deferred payments and installment options allowing reimbursement bodies to manage budget impact during the early access phase
- Outcomes-based rebates negotiated following clinical and economic assessments can be applied to patients treated during the early access period
- Robust training for treating institutions on administration and follow-up care
- Access to RESTORE, a global registry of patients who have been diagnosed with SMA that draws upon existing country registries
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Novartis Media Relations Central media line: +41 61 324 2200 E-mail:?media.relations@novartis.comAnja von Treskow Novartis Global External Communications +41 61 79 392 8697(mobile) Anja.von_treskow@novartis.com Eric Althoff Novartis External Communications +1 646 438 4335 (mobile) eric.althoff@novartis.com |