FDA Approves Evrysdi in Pre-symptomatic Newborns with SMA

The FDA has approved a label extension for Evrysdi (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment.

Evrysdi is now approved to treat SMA in children and adults of all ages. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

In a small study of six patients, all were able to sit after one year of treatment, 67% could stand and 50% of infants could walk independently. All infants were alive at 12 months without permanent ventilation.

Richard S. Finkel, M.D.

“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” Richard S. Finkel, M.D., RAINBOWFISH principal investigator, said in a press release. He is director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital. “With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”

As part of the label extension, the Evrysdi prescribing information has also been updated to include recent two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrate long-term efficacy and safety in symptomatic infants with Type 1 SMA. The study enrolled babies aged 1-7 months and after two years of treatment with Evrysdi, 60% of infants were able to sit without support for 5 seconds, 40% for 30 seconds and 28% of infants were able to stand. There were no treatment-related adverse events leading to withdrawal. The most common adverse reactions were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting and cough.