25th July 2025

Tablet form of risdiplam (Evrysdi) approved in the UK. Roche working to get supplies for eligible patients by mid -August.

The UK Medicines and Health Products Regulatory Agency (MHRA) have now approved the room-temperature stable tablet formulation of Evrysdi.

The tablet formulation is approved for people living with:

• SMA Type 1, Type 2 or Type 3 or
• one to four SMN2 copies, who are 2 years of age or older with a body weight of 20 kg (44 lbs) or more.

They must be able to swallow without the use of a feeding tube.

For full details please see the Summary of Product Characteristics >.

The approval of the 5 mg tablet provides an alternative formulation for eligible individuals. The Evrysdi tablet:

• can either be swallowed whole or dispersed in water
• can be taken with or without food
• does not require refrigeration
• can be stored at room temperature.

In response to SMA community feedback, the tablet was developed to support further flexibility and choice for people living with SMA.

What happens next?

The MHRA approval means that the 5 mg Evrysdi tablet is approved for use in the UK. Access to the tablet formulation will be the same as with the oral solution.

• In England, Northern Ireland and Wales eligible individuals can receive the Evrysdi tablet under the existing managed access agreement (MAA).
• In Scotland people are able to access Evrysdi via the NHS.

This new formulation will be automatically incorporated.

Roche is working to get supply available to patients in the UK as soon as possible. It is likely stock will be available by mid-August. The Evrysdi oral solution will remain available for those who are not eligible for the tablet formulation and those who may prefer the oral solution.

Evidence supporting the approval

The approval of the Evrysdi tablet was based on the results of a bioequivalence study (NCT04718181) which demonstrated that the tablet formulation and the oral solution are bioequivalent. This means that the tablet provides the same Evrysdi exposure. It is expected to have the same efficacy and safety profile as the oral solution.

Thanks to the SMA Community

Roche has asked us to pass on this message to the SMA Community:

"We would like to thank the SMA community  once again for your continued partnership, which enables milestones like this one. Without your trust, advice, and participation in clinical research, none of this would be possible."

Further information from Roche >

Previous Updates

12th June 2025

On 4th June, Roche announced that the European Commission (EC) approved a label extension for Evrysdi® (risdiplam). This now includes a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). The 5mg tablet (approx. 6.5mm):

• can be either swallowed whole or dispersed in water,
• can be taken with or without food
• does not require refrigeration, when stored at room temperature.

Administered at home, Evrysdi is the only non-invasive disease modifying treatment available for people living with SMA.

Roche’s Press Release re: EC approval >

12th February 2025

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

2023 – 2024

Study to develop a Tablet form of Risdiplam

A Phase 1 study (NCT04718181) was completed in 2023.

The study was designed to assess how well a tablet of 5mg risdiplam that could be swallowed whole or dispersed in a small amount of bottled water would work.

BABE (Bioavailability and bioequivalence)results were presented at the SMA Europe congress in February 2024. They showed that, whether swallowed whole or dispersed:

• The tablet resulted in the same concentration of risdiplam in the body as the currently available oral solution
• The tablets may be given with or without food. Food had no effect on how well the tablet were absorbed. This was similarly demonstrated for the oral solution.
• No new safety signals were identified.

In the first half of 2024, Roche planned to apply to the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) for the tablet formulation to be considered for licencing.