24 May 2019 - ICER’s update reflects the gene therapy’s new clinical data and FDA label.

In light of additional data from ongoing trials of onasemnogene abeparvovec (Zolgensma, Novartis/AveXis), as well as the treatment’s final FDA label and just-announced launch price, ICER today published an addendum to its Final Evidence Report on treatments for spinal muscular atrophy (SMA).

Since the publication of ICER’s Final Evidence Report on April 3, 2019, early results of a Phase III single arm trial (SPR1NT) have been encouraging for the use of Zolgensma in patients with pre-symptomatic SMA. ICER’s Final Evidence Report had included a hypothetical scenario analysis that modelled the cost-effectiveness of Zolgensma assuming a level of efficacy in the pre-symptomatic population that may be similar to what is now observed in early results from the SPR1NT trial:

• To reach commonly cited cost-effectiveness thresholds of $100,000 to $150,000 per quality-adjusted life year (QALY) gained, a value-based price benchmark for Zolgensma would be between $1.1 million to $1.9 million per treatment.
• To reach the alternative thresholds of $100,000 to $150,000 per life year gained (LYG), a value-based price benchmark for Zolgensma would be between $1.2 million to $2.1 million.

Read ICER announcement