Genentech announced that the US Food and Drug Administration (FDA) approved the use of risdiplam (Evrysdi®) in babies less than 2 months old with spinal muscular atrophy (SMA). The approval comes after encouraging interim efficacy and safety data were obtained on newborns in the RAINBOWFISH study.

The study showed that most presymptomatic babies undergoing treatment with risdiplam achieved important milestones after 12 months of therapy, including sitting and standing with half walking.

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


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“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.”

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Risdiplam is a survival motor neuron 2 splicing modifier that is taken daily in liquid form. It is designed to increase and sustain the production of the SMN protein in the central nervous system and other tissues, thereby supporting healthy motor neurons and motor development.

The new approval includes updated data from parts 1 and 2 of the FIREFISH trial, which showed that risdiplam had long-term efficacy and safety in symptomatic infants with type 1 SMA, helping them achieve sitting and standing milestones that infants without treatment do not achieve in the natural course of SMA.

Risdiplam is now approved for both pediatric and adult patients with SMA of any age. It is being evaluated in 5 ongoing clinical trials in patients with SMA.

Reference

FDA approves Genentech’s Evrysdi (risdiplam) for use in babies under two months with spinal muscular atrophy (SMA). News release. Genentech; May 31, 2022.