24 May 2019 - Annualised 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 spinal muscular atrophy therapy.

AveXis, a Novartis company, today announced innovative access programs for Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of paediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. AveXis is working closely with payers to offer pay-over-time options up to 5 years and outcomes-based agreements up to 5 years, as well as providing a patient program to support affordability and access.

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)
• 50% below 10-year treatment costs for genetic paediatric ultra-rare diseases (estimated at USD 4.4 million to USD 5.7 million)
• 50% below the ICER ultra-rare disease cost-effectiveness threshold; Zolgensma pricing places it at approximately USD 250,000 per quality-adjusted life-year gained

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