ICER investigating spinal muscular atrophy drugs

21 December 2018

The USA’s Institute for Clinical and Economic Review (ICER) yesterday released a Draft Evidence Report assessing the clinical effectiveness and value of Spinraza (nusinersen) from Biogen (Nasdaq: BIIB) and Zolgensma (onasemnogene abeparvovec), from Novartis (NOVN: VX) and its subsidiary AveXis, for the treatment of spinal muscular atrophy (SMA).

Spinraza was approved in 2016 for treatment of SMA in both children and adults. Zolgensma is a gene therapy that has been studied in infants with Type I SMA. AveXis announced the Biologics License Application had been accepted by the US Food and Drug Administration early this month, and an FDA decision is expected in May 2019.

While the report judged both as priced too high to be cost-effective at typical thresholds, cost per incremental quality-adjusted life year (QALY) for Zolgensma at a hypothetical $2mm price were markedly lower than for Spinraza. If confirmed in a final report in March, these findings could offer support for Novartis in pricing and access negotiations for Zolgensma ahead of 2019 estimated approval, noted Leerink Research.

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