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).

The new tablet formulation can either be swallowed whole or dispersed in water. It can be stored at room temperature.  The tablet offers the same demonstrated efficacy and safety as the currently available oral solution.

See: Roche’s Media Release >

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.