Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA).
SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies.
The drug first won FDA approval in 2020 for adults and children at least two months old.
The label extension was based on interim efficacy and safety data from the RAINBOWFISH study in newborns.
That data demonstrated the majority of pre-symptomatic babies who received Evrysdi achieved developmental milestones related to sitting and standing. In addition, half of the babies were walking after 12 months of treatment.
“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 Dr. Richard Finkel, 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.”
Evrysdi modulates the survival of motor neuron-2 (SMN2) protein.
More than 5,000 patients have been treated with the drug to date, including babies, children and adults.
Spinal muscular atrophy results from the homozygous deletion or mutations in survival motor neuron-1 (SMN1).
Extra copies of the SMN2 gene do not cause spinal muscular atrophy, but they modify the severity of the disorder, according to NIH.
Filed Under: Neurological Disease