NICE recommends Roche risdiplam for spinal muscular atrophy

CompilationFan Dongdong

NICE UK has released draft guidelines recommending Roche risdiplam (Evrysdi) as part of a managed access agreement (MAA) for the treatment of the rare genetic disease spinal muscular atrophy (SMA)

It also means risdiplam, a revolutionary new treatment, will be introduced to NHS Medicare UK to help tackle the leading genetic cause of death in infants and young children

The MAA started as a special agreement between NHS England, NHS Improvement and Roche to allow people to be treated with risdiplam while more data was collected to address uncertainty about the drug by the independent UK NICE committee, and later by NICE decides whether to recommend the drug for routine use in the UK NHS and updates guidelines

In August this year, Roche announced that the U.

This UK NICE draft guideline recommends risdiplam for clinical diagnosis of type 1, 2 or 3, or two with presymptomatic spinal muscular atrophy and 1 to 4 copies of the survival motor neuron 2 (SMN2) gene Children with spinal muscular atrophy aged 6 months and older

The drug has shown good efficacy in the pivotal Firefish study (in children 2 to 7 months) and the Sunfish study (in patients 2 to 25 years)

In addition, 90% of children survived to 12 months without mechanical ventilation, and survived beyond 15 months

This NICE recommendation for risdiplam for the treatment of spinal muscular atrophy is especially important for those patients with spinal muscular atrophy who were unable to receive the two previous treatments (Spinraza and Zolgenasma)

Reference source: NICE recommends risdiplam for spinal muscular atrophy