Appraisal of risdiplam in Scotland further delayed

Comcerns over delays with Roche’s submission for risdiplam (also known as Evrysdi)

SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise their concerns about the further delays with processing Roche’s submission for risdiplam (also known as Evrysdi). Though we understand this delay is due to the staff and appraisal committees’ workloads, the Scottish SMA community is already facing considerable difficulties because, although Spinraza (nusinersen) has been accepted for restricted use within NHS Scotland for both children and adults, there is as yet still no delivery of this treatment to adults. Additionally, due to Spinraza’s intrathecal method of delivery, it is not a clinically safe treatment for the majority with severe scoliosis / spinal surgery. SMA is a progressive condition and every day counts. We therefore urge the SMC to consider expediting the appraisal process for risdiplam on the basis that for a significant proportion of the SMA community there is no other alternative treatment and indeed it may be a preferred option for some already treated with Spinraza