Zolgensma® – Access Programs for US Payers and Families

Basel, May 24, 2019 – AveXis, a Novartis company, today announced innovative access programs for Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Annualized cost of Zolgensma is USD 425,000 per year for 5 years: 50% less than multiple established value-based pricing benchmarks including the 10-year current cost of chronic SMA therapy[1]

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Liz

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  1. The current 10-year cost of chronic therapy, which is given over the patient’s lifetime, can often exceed USD 4 million in just the first 10 years of a young child’s life.[1] In addition, that therapy stops working if treatment is stopped.[2] Zolgensma is expected to save costs in the healthcare system compared to chronic treatment for the treatment and care of SMA.[3] The wholesale acquisition cost of Zolgensma of USD 2.125 million is:

    50% of the 10-year cost of current chronic SMA treatment (estimated at USD 4.1 million)[1]
    50% below 10-year treatment costs for genetic pediatric ultra-rare diseases (estimated at USD 4.4 million to USD 5.7 million)[4]*
    50% below the ICER ultra-rare disease cost-effectiveness threshold; Zolgensma pricing places it at approximately USD 250,000 per quality-adjusted life-year (QALY)[5]

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