Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.

In December 2023, the Medicines and Healthcare products Regulatory Agency approved a licence extension for risdiplam to include people of all ages. We updated the recommendation and information in section 2 to account for this extension to the marketing authorisation.

Is this guidance up to date?

Next review: In December 2021 we decided that we will review the evidence when the results of the FIREFISH and SUNFISH trials are published.

Commercial arrangement

There is a managed access agreement, which includes a commercial access agreement for risdiplam. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact welwyn.rx_bdop@roche.com for details.

In December 2023, we published a variation to the managed access agreement to align with the guidance update. This extends eligibility to people of all ages (previously 2 months and over).

Guidance development process

How we develop NICE technology appraisal guidance

Your responsibility

The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.

All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the Yellow Card Scheme.

Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.

Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible.

  • National Institute for Health and Care Excellence (NICE)