Onasemnogene abeparvovec for treating spinal muscular atrophy – guidance (HST15)

NICE recommend onasemnogene abeparvovec for 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of type 1 SMA in babies under set restrictions.

SPS commentary:

The restrictions are:

    The patient is 6 months or younger or
    They are aged 7 to 12 months, and their treatment is agreed by the national multidisciplinary team.

It is only recommended if permanent ventilation for more than 16 hours per day or a tracheostomy is not needed

For babies aged 7 to 12 months, the national multidisciplinary team should develop auditable criteria to enable onasemnogene abeparvovec to be allocated to babies in whom treatment will give them at least a 70% chance of being able to sit independently.

It is also recommended as an option for treating presymptomatic 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene in babies.

It is only recommended if the company provides it according to the commercial arrangement and the conditions in the managed access agreement are followed.

National Institute for Health and Care Excellence