AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy (SMA) - Seite 4

• 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 SMA registry of patients who have been diagnosed with SMA that draws upon existing country registries

About Spinal Muscular Atrophy
SMA is the leading genetic cause of infant death.^2,3 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.⁵
In Europe each year, approximately 550–600 infants are born with SMA.^6,7 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.² It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.⁸ This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.⁹ More than 30% of patients with SMA Type 2 will die by age 25.¹⁰

Novartis will conduct a conference call with investors to discuss this news release on Monday, March 30, 2020 at 3 p.m. Central European Time and 9 a.m. Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar.

About Zolgensma (onasemnogene abeparvovec)
Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma represents the first approved therapeutic in the company’s proprietary platform to treat rare, monogenic diseases using gene therapy.⁶ Approximately 400 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program in the U.S. AveXis is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada and Australia in late 2020 or early 2021.⁶