Roche has received approval from the US Food and Drug Administration (FDA) for Evrysdi (risdiplam) to be used in babies aged below two months with spinal muscular atrophy (SMA). 

A survival motor neuron 2 (SMN2) splicing modifier, Evrysdi is intended for treating SMA caused by mutations in chromosome 5q that result in a deficiency of the SMN protein. 

The FDA approved label extension for the therapy to include patients of this age group based on interim efficacy and safety findings from the open-label, single-arm, multicentre RAINBOWFISH clinical trial in newborns.

The trial analysed the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies from their birth to six weeks of age with genetically diagnosed SMA that have not shown any symptoms.

Trial data showed that most of the pre-symptomatic babies who received Evrysdi attained crucial milestones such as sitting and standing, with half of the babies walking following 12 months of treatment. 

Among the infants with two or three copies of the SMN2 gene, all of them could sit following one year of Evrysdi treatment. 

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In addition, 67% and 50% of trial subjects could stand and walk, respectively. 

All the babies were alive for one year without needing permanent ventilation.

As part of the latest development, the Evrysdi prescribing information was revised to include the latest two-year pooled findings from Parts 1 and 2 of the FIREFISH trial, which showed durable safety and efficacy of the therapy in symptomatic infants with Type 1 SMA. 

Evrysdi is currently approved in the US for treating children and adults with SMA.

Roche Global Product Development head and chief medical officer Levi Garraway said: “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. 

“Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults.”

The latest development comes after the UK Medicines and Healthcare products Regulatory Agency approved Roche’s faricimab (Vabysmo) for wet age-related macular degeneration (wet AMD) and impairment in vision due to diabetic macular oedema (DMO).