Improvement in motor function with Evrysdi® in presymptomatic SMA

Approved for spinal muscular atrophy (SMA), the efficacy of Evrysdi® in presymptomatic forms remained uncertain. In the international RAINBOWFISH open-label trial, 26 genetically diagnosed infants received Evrysdi® for two years within the first six weeks of life before the onset of symptoms.

  • After 12 months of daily oral treatment, 81% of infants could sit unaided for 30 seconds, 54% could stand unaided, and 42% could walk unaided.
  • The 23 infants who completed 24 months of treatment did not require permanent respiratory or nutritional support.
  • Nine non-serious adverse events related to treatment were reported in seven infants.

Larger controlled studies with longer follow-up will be necessary.

 

Risdiplam in Presymptomatic Spinal Muscular Atrophy. Finkel R, Servais L, Dmitry Vlodavets D et al. N Engl J Med. 2025 Aug 14:671-682.

 

On Clinicaltrials.gov: NCT03779334