FDA Expands Approval of SMA Drug Evrysdi to Include Infants Under Two Months Old

The FDA has granted an expanded approval for Roche’s oral solution drug Evrysdi (risdiplam) to include pediatric patients under two months old with the rare genetic disorder spinal muscular atrophy (SMA).

The agency based the expanded approval on interim data from a study that enrolled infants aged from birth to six weeks old at first dose who were genetically diagnosed with SMA but not yet symptomatic.

The majority of presymptomatic infants treated with Evrysdi achieved key milestones, including sitting and standing, with half of the participants walking after 12 months of treatment, the company said.

In August 2020, the FDA approved Evrysdi for pediatric and adult SMA patients aged two months and older.