Evrysdi Gets FDA Approval for SMA Babies Under 2 Months Old

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old.

Evrysdi, an oral therapy that works by increasing the levels of the survival motor neuron (SMN) protein that is missing in SMA, had previously been approved to treat all types of SMA only for patients ages 2 months and up.

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults,” Levi Garraway, MD, PhD, said in a press release.

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Garraway is the chief medical officer and head of global product development at Genentech, a subsidiary of Roche, which markets Evrysdi and helped develop the therapy in collaboration with PTC Therapeutics and the SMA Foundation.

“The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” Garraway said. “We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”

The FDA’s extended approval was supported by interim data from a Phase 2 clinical trial called RAINBOWFISH (NCT03779334), which is testing Evrysdi in babies diagnosed with SMA who start treatment before displaying any overt symptoms.

Data from six babies with two or three copies of the SMN2 gene showed that all of the treated babies were alive, did not need permanent ventilation, and were able to sit unassisted after one year of treatment. Four of the children could stand at one year, and three of them were able to walk.

“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, director of the experimental neuroscience program at St. Jude Children’s Research Hospital and principal investigator of the RAINBOWFISH study.

“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,” Finkel added.

The label extension also includes the addition to Evrysdi’s prescribing information of new data from the Phase 2/3 FIREFISH study (NCT02913482), which enrolled infants with type 1 SMA who started treatment with Evrysdi in the first months of life.

Data showed that, after two years on Evrysdi, most of the children could sit unassisted for at least five seconds, and more than 1 in 4 were able to stand.

There were no treatment-related adverse events leading to withdrawal. The most common adverse events reported in the study included upper and lower respiratory tract infections, constipation, vomiting, and cough.