Spinal Muscular Atrophy

Treatments of Interest:

• onasemnogene abeparvovec (Zolgensma®, Novartis AG/AveXis)
• nusinersen (Spinraza®, Biogen)

Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease. In its most common form, Type I SMA (~60% of cases), symptoms begin in infancy. Affected children never sit up, and they rapidly develop difficulty swallowing and breathing, eventually requiring feeding tubes and mechanical ventilation. Without treatment, children typically die by age 2.

The independent appraisal committee noted family testimony in votes confirming broad benefits of both treatments; however, the committee voted unanimously that the price for Spinraza is too high to align fairly with these benefits, and urges fair pricing for emerging gene therapy Zolgensma to support sustainable access to innovation.

ICER’s Chief Medical Officer David Rin, MD, MSc stated:

“Both Spinraza and Zolgensma dramatically improve the lives of children with SMA and that of their families. However, the current price of Spinraza far exceeds common thresholds for cost-effectiveness. The price of Zolgensma is not yet known, but there has been public discussion of prices above commonly accepted cost-effectiveness thresholds as well. These treatments will be covered by US insurers regardless of the pricing, but the ripple effect of pricing decisions like these threatens the overall affordability and sustainability of the US health system.”

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