PTC Therapeutics, Inc. (NASDAQ: PTCT) confirmed that US FDA has authorized a label extension for Evrysdi (risdiplam) to include infants under 2 months old with spinal muscular atrophy (SMA). Evrysdi is a treatment for SMA that works by raising and sustaining the production of the SMN protein, which is located all over the body and is essential for maintaining healthy motor neurons and movement.
PTC’s splicing platform served as the foundation for Evrysdi. Evrysdi is promoted by Roche and Genentech, a Roche Group company, in the United States. As part of a partnership with the SMA Foundation and PTC Therapeutics, Roche is leading the clinical development of Evrysdi. The approval of Evrysdi is based on interim efficacy and safety results from the RAINBOWFISH newborn research.
The research found that pre-symptomatic neonates treated with Evrysdi completed important milestones like sitting and standing, as well as half-walking, after 12 months of treatment. At the age of 12 months, all of the infants were still alive. The label extension of Evrysdi to encompass pre-symptomatic infants is a key next step in treating babies with SMA as early as possible.
Furthermore, it has been discovered that all of the babies who received Evrysdi treatment were able to reach developmental milestones in the same time frame as infants who did not have SMA. PTC Therapeutics is ecstatic that these children will have access to such a life-changing medication for SMA.