Nusinersen
Nusinersen can be used as an option to treat 5q spinal muscular atrophy (SMA), only if:
it has not been successfully treated with onasemnogene abeparvovec (see section 3.10)
nusinersen is not used with risdiplam, and
the company provides nusinersen according to the commercial arrangement.
Stop nusinersen if there is sustained deterioration across a range of clinical measures and the treatment is not providing benefit. Assess whether the person is benefitting from treatment based on clinical judgement of their individual needs.
Risdiplam
Risdiplam can be used as an option to treat 5q SMA type 1, 2 or 3 or with 1 to 4 SMN2 copies, only if:
it has not been successfully treated with onasemnogene abeparvovec (see section 3.10)
risdiplam is not used:
with nusinersen
for presymptomatic SMA when onasemnogene abeparvovec is suitable, and
the company provides risdiplam according to the commercial arrangement.
Stop risdiplam if there is sustained deterioration across a range of clinical measures and the treatment is not providing benefit. Assess whether the person is benefitting from treatment based on clinical judgement of their individual needs.
This recommendation is not intended to prevent risdiplam being used as a bridging therapy until treatment with onasemnogene abeparvovec is possible.
Nusinersen and risdiplam must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. NHS England has agreed to provide funding from routine commissioning budgets so nusinersen and risdiplam will be funded from 14 May 2026.
There is enough evidence to show that nusinersen and risdiplam provide benefits and value for money, so they can be used routinely across the NHS in this population.
NICE has produced tools and resources to support the implementation of this guidance.
This evaluation reviews the evidence for nusinersen and risdiplam for SMA (NICE technology appraisal guidance TA588 and TA755). It also reviews new evidence collected during the managed access period, which includes evidence from clinical trials and from people having treatment in the NHS in England.
Usual treatment for presymptomatic or type 1 SMA is onasemnogene abeparvovec. When this is not suitable, and for other types of SMA, usual treatment is best supportive care. The managed access agreements specified that people could not have nusinersen or risdiplam if they had already had successful treatment with onasemnogene abeparvovec.
Clinical trial evidence shows that, compared with best supportive care, nusinersen and risdiplam improve motor function and increase how long people live. They have not been directly compared with onasemnogene abeparvovec in a clinical trial. The results of indirect comparisons are highly uncertain. But they suggest that nusinersen and risdiplam have similar benefits to onasemnogene abeparvovec.
This evaluation considered the condition's severity and its effect on quality and length of life.
For presymptomatic SMA, the most likely cost-effectiveness estimate for risdiplam is only within what NICE considers an acceptable use of NHS resources when onasemnogene abeparvovec is not suitable.
For all other SMA types, the most likely cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources. So, nusinersen and risdiplam can be used.