Roche today announced that the US Food and Drug Administration (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 in the US to treat SMA in children and adults of all ages, Roche said in a statement.
Of the babies with two or three copies of the SMN2 gene (n=6), 100 per cent were able to sit after one year of treatment with Evrysdi, 67 per cent could stand and 50 per cent of infants could walk independently. All infants were alive at 12 months without permanent ventilation, the statement informed.
It further noted that 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-I SMA. The study enrolled babies aged one-to-seven months and after two years of treatment with Evrysdi at the recommended dose (n=58), 60 per cent of infants were able to sit without support for five seconds, 40 per cent for 30 seconds and 28 per cent of infants were able to stand.
The statement also said that without treatment, infants do not achieve these milestones in the natural history of the disease. 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.
Evrysdi is approved in 81 countries and the dossier is under review in further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics, the statement concluded.