Skip to content
Make a donation
Make a donation

Risdiplam (Evrysdi)

Risdiplam (Evrysdi)

FDA approves Roche’s Evrysdi, (risdiplam) tablet as first and only tablet for Spinal Muscular Atrophy (SMA) 

On 12 February 2025 – Roche announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA).  

The new tablet formulation may provide greater freedom and independence for people with SMA thanks to simplified dose administration. It can either be swallowed whole or dispersed in water and it can be stored at room temperature.  The tablet offers the same demonstrated efficacy and safety as the currently available oral solution. 

Find more information on the tablet here  and watch this space for updates on access to the tablet in the UK.  

Last checked: 18th December 2024

What it is, How it works, and Other FAQs

Risdiplam is a small molecule drug that targets the ‘back up’ survival motor neuron 2 (SMN2) gene to produce more SMN protein.

Read more>

UK Access Now – England

Risdiplam is funded by the NHS via a Managed Access Agreement.

Read more>

UK Access Now – Scotland

The NHS in Scotland funds risdiplam.

Read more>

UK Access Now – Wales and Northern Ireland

In Wales and Northern Ireland, risdiplam is funded under the same terms as for England’s Managed Access Agreement.

Read more>

Risdiplam Clinical Trials and Their Results

Risdiplam has been tested in human clinical trials since 2015. It has been consistently shown to have a well-understood safety profile. It has been well-tolerated in several different trials across 5q SMA populations.

When compared to placebo or no treatment, risdiplam has been shown to increase the availability of SMN protein in many people. This has led to clinically meaningful improvements in muscle function. Several trials of risdiplam are still ongoing.

Read more>

Our Advocacy and Progress Towards Risdiplam Access in the UK

We have worked hard with the SMA Community, Muscular Dystrophy UK, TreatSMA and clinicians to advocate for access, and will continue to work towards access throughout the UK.

Read more>