UK – NICE recommends risdiplam for spinal muscular atrophy

NICE has published draft guidance recommending risdiplam (Evrysdi) as part of a managed access agreement (MAA) for the treatment of the rare genetic disorder spinal muscular atrophy (SMA). The NHS will introduce the revolutionary new treatment, tackling the leading genetic cause of death among babies and young children. Around 1500 patients in England are expected to benefit from the drug, which can be taken as a syrup at home.

The MAA is a special agreement between NHS England, NHS Improvement, and the company Roche, allowing people to access treatment with risdiplam while more data is collected, addressing the uncertainties identified by the independent NICE committee. NICE will then decide whether to recommend the drug for routine use on the NHS and update guidance. Risdiplam will be available through the MAA until then…