12th June 2025

On 4th June, Roche announced that the European Commission (EC) has 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.

We asked Roche UK what plans they have to apply for this tablet to be made available in the UK.  They confirmed that:

• They have submitted their license application for the risdiplam tablet formulation to the Medicines and Healthcare products Regulatory Agency (MHRA).
• They do not currently have any accurate information regarding the MHRA  timelines.
• Once MHRA approval is granted, the risdiplam tablet would not need to undergo review by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC).

• Roche’s Press Release re: EC approval >

Previous Updates

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.