FDA Approves Expanded Indication of Risdiplam for Spinal Muscular Atrophy to Include Infants Under Age 2 Months

The Food and Drug Administration (FDA) has approved an expanded indication for risdiplam (Evrysdi; Genentech, South San Francisco, CA) to include neonates (age 0-8 weeks) with presymptomatic, genetically confirmed spinal muscular atrophy (SMA). 

The expanded indication is supported by data from the RAINBOWFISH study (NCT03779334) showing presymptomatic neonates with SMA who were treated with risdiplam achieved key milestones (eg, sitting and standing), with half walking a12 months of treatment. Participants who had 2 or 3 copies of the survival of motor neuron 2 (SMN2) gene (n=6) survived and were able to sit after a year of treatment. Standing and independent walking were achieved by 67% and 50%, respectively, after 12 months of treatment. 

“. . .early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, MD, RAINBOWFISH principal investigator and 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.”
 
The most common adverse event found during treatment were constipation, cough, lower respiratory tract infection, upper respiratory tract infection, and vomiting.
 
Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of Global Product Development said, “Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from presymptomatic newborns to older adults. We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”