Posted by Michael Wonder on 02 Jun 2021
NICE draft guidance does not recommend risdiplam for treating spinal muscular atrophy
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for treating the rare genetic disorder spinal muscular atrophy.
The Appraisal Committee agreed that the clinical trials demonstrate that risdiplam meaningfully improves motor function for people with type 1, 2 and 3 SMA, but the long term benefits are uncertain.
