Novartis' AveXis unit views ICER findings on its Zolgensma

Swiss pharma giant Novartis’ (NOVN: VX) subsidiary AveXis has reacted to the release on Friday of a  report assessing the likely cost-effectiveness of the company’s investigational spinal muscular atrophy (SMA) drug Zolgensma (onasemnogene abeparvovec- xioi), as well as slamming the price of Biogen’s already marketed Spinraza (nusinersen).

“We appreciate that the Institute for Clinical and Economic Review (ICER) delivered a thorough analysis that clearly establishes the significant medical, economic and societal burdens faced by families affected by spinal muscular atrophy (SMA). We believe that ICER’s model captures the clinical progression of SMA Type 1 and are pleased that the draft report highlights the milestones achieved by patients receiving Zolgensma, who otherwise would have experienced rapidly progressing and often deadly disease,” the AveXis statement noted.

It said the company is pleased that the ICER acknowledges cost-effectiveness thresholds up to $500,000 per quality-adjusted life-year (QALY) gained, which both the rare disease community and various governmental bodies have suggested is the appropriate standard to protect vulnerable populations and allow access to innovative, transformational therapies for a small number of patients, said AveXis.