Study shows significant improvement of survival in infants with SMA
pharmafile | July 29, 2021 | News story | | sma
Results from the Evrysdi part 2 global FIREFISH study show significant improvement in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
The study showed treatment with Evrysdi helped babies stay free of permanent ventilation, sit without support, and improve across a range of motor milestones.
The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least five seconds by month 12, a milestone not seen in the natural course of the disease.
Additionally, after treatment, 93% of infants were alive and 85% were free from permanent ventilation.
Professor Laurent Servais, FIREFISH investigator, said: “Without treatment, babies with Type 1 SMA are unlikely to survive beyond two years of age.
“Important motor milestones, such as sitting, rolling over, and swallowing, are the fundamental building blocks that can help these babies achieve optimal outcomes with Evrysdi, potentially reducing the need for ventilation and increasing the rate of survival.”
The study also met one of its secondary endpoints with 78% of infants classified as HINE-2*** responders, which evaluated motor function through head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking. Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.
Levi Garraway, Genentech’s Chief Medical Officer and head of Global Product Development, said: “These data published in the New England Journal of Medicine validate results from Part 1 of the FIREFISH study that showed Evrysdi can help babies with SMA reach the significant milestone of sitting without support for at least five seconds.
“These results have been further confirmed in the recently presented 24-month data showing Evrysdi continued to improve motor function, doubling the number of babies able to sit without support from month 12. We will continue to work closely with governments and the SMA community to bring Evrysdi to as many people as possible.”
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.
Lilly Subbotin
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