SMA drug now approved for patients of all ages

Genentech announced that the FDA approved a label extension for Evrysdi to include infants younger than 2 months with spinal muscular atrophy.

The approval is based on interim efficacy and safety data from the RAINBOWFISH study, which showed that in newborns with two or three copies of the SMN2 gene (n = 6), 100% were able to sit after 1 year of treatment with Evrysdi (risdiplam, Genentech/Roche Group), 67% were able to stand, and 50% could walk independently. All infants were alive at 12 months without permanent ventilation, according to a company press release.

“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 Finkel, MD, RAINBOWFISH principal investigator and director of the experimental neuroscience program at St. Jude Children’s Research Hospital, said in the release.

Evrysdi is now approved to treat SMA in children and adults of all ages.

As part of the label extension, prescribing information for Evrysdi was updated to include recent 2-year pooled data from parts 1 and 2 of the FIREFISH study, which demonstrated long-term efficacy and safety in symptomatic infants aged 1 to 7 months (n = 58) with type 1 SMA. After 2 years of treatment at the recommended dose, 60% of participants were able to sit without support for 5 seconds, 40% for 30 seconds, and 28% were able to stand. Without treatment, infants do not achieve these milestones in the natural course of the disease, the company said in the release.

No treatment-related adverse events led to withdrawal, and the most common adverse reactions included upper respiratory tract infection (including nasopharyngitis and rhinitis), lower respiratory tract infection (including pneumonia and bronchitis), constipation, vomiting and cough, the company said.

“The priority review and subsequent approval of Evrysdi for babies under 2 months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of global product development, said in the release.