Positive five-year data for Roche’s Evrysdi in SMA

Swiss pharma giant Roche (ROG: SIX) on Friday announced new five-year data confirming the sustained efficacy and safety profile of Evrysdi (risdiplam) in children with Type 1 spinal muscular atrophy (SMA) from the open-label extension of the pivotal FIREFISH study.

Marketed by Roche and its subsidiary Genentech, the treatment has been developed in collaboration with PTC Therapeutics (Nasdaq: PTCT) and the SMA Foundation. Analysts have previously forecast that risdiplam could bring in up to $2 billion at peak. Now approved in more than 100 countries, the drug generated 1.4 billion Swiss francs ($1.56 billion) sales in 2023.

By the end of year five, 91% of children treated with Evrysdi were alive, 81% were alive without permanent ventilation and the majority were able to sit without support for at least 30 seconds (59%). At the end of year five, seven children were able to stand, three with support, four unaided and six could walk with support.