PTC Therapeutics, Inc. (NASDAQ: PTCT) disclosed that it has received priority review of a supplemental new drug application (sNDA) for Evrysdi by the U.S FDA to increase the indication to include pre-symptomatic infants under 2 months old with spinal muscular atrophy (SMA). Evrysdi would be the first medicine administered at home for pre-symptomatic babies with SMA if authorized.
Interim data from the RAINBOWFISH study has been included in the sNDA submission, showing that 80 percent of pre-symptomatic infants with SMA treated with Evrysdi for at least 12 months accomplished motor benchmarks like sitting without assistance, rolling, crawling, standing unassisted, and strolling independently.
Furthermore, interim data from the RAINBOWFISH study has been included in the sNDA submission. The data highlighted that 80 percent of pre-symptomatic newborns with SMA treated with Evrysdi for at least 12 months accomplished motor benchmarks. They were able to sit without assistance, crawl, stand unassisted, and stroll independently.
In addition, the award of the Priority Review for Evrysdi understands the significant need to cure babies with SMA as early as possible. The company is excited that such a life-changing treatment for patients living with SMA came from its splicing platform. Moreover, Evrysdi is meant to treat SMA by increasing and keeping the production of the SMN protein, which is discovered throughout the body and is vital for maintaining healthy motor neurons and movement.