Posted by Michael Wonder on 22 Aug 2018
ICER posts draft scoping document on AVXS-101 and nusinersen for spinal muscular atrophy
22 August 2018 - Document open to public comment until 7 September 2018.
The ICER has posted a draft scoping document outlining a planned review of the comparative clinical effectiveness and value of AVXS-101 (AveXis) and nusinersen (Spinraza, Biogen) for the treatment of spinal muscular atrophy (SMA).
Nusinersen was approved in 2016 for treatment of SMA in both children and adults. AVXS-101 is a gene therapy currently being studied for use in infants with SMA, and an FDA decision is expected in the first quarter of 2019. ICER’s report on these therapies will be the subject of a March 2019 meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.
