PTC Therapeutics, Inc. announced that the FDA has approved a label extension for Evrysdi® (risdiplam) to include infants under 2 months old with spinal muscular atrophy (SMA).
"The label extension for Evrysdi to include pre-symptomatic infants is the critical next step for intervening as early as possible in treating babies with SMA," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "Evrysdi treatment allowed almost all of the babies to achieve developmental milestones in a similar timeframe as infants who don't have SMA. We are proud that such a transformative treatment for SMA will be available to these infants."
The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi achieved key milestones, such as sitting and standing, with half walking after 12 months of treatment. All infants were alive at 12 months without permanent ventilation.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi was based on PTC's splicing platform. Evrysdi is marketed by Roche and in the United States by Genentech, a member of the Roche Group. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
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