SUNFISH is assessing the safety, tolerability, and efficacy of Evrysdi compared with a placebo in people with type 2 or type 3 SMA, ages 2 to 25.
Two children also were able to stand without support.
The study already met its primary goal, with almost 30% of enrolled infants able to sit without support for five seconds after one year of treatment.Ī number of infants in the trial have achieved other clinically meaningful milestones, including gaining upright head control and being able to roll from side to side. The FIREFISH trial is evaluating Evrysdi’s safety, tolerability, efficacy, and pharmacological properties in infants ages 1 to 7 months, with symptomatic type 1 SMA. Two ongoing clinical trials - FIREFISH ( NCT02913482) and SUNFISH ( NCT02908685) - supported the applications filed in both the U.S. Access to a home administered therapy is particularly important during the COVID-19 pandemic,” Peltz said. “The strong efficacy, excellent safety profile, and convenient oral formulation of Evrysdi are so important to the SMA community. It also allows patients to take the medicine at home, a key consideration during the ongoing COVID-19 pandemic. This delivery method distributes Evrysdi throughout the body, where it acts on the motor neurons of the central nervous system (composed of the brain and spinal cord), as well as muscles and other tissues.
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