RARE Daily

FDA Grants Evrysdi Priority Review Based on Results from Treating Pre-Symptomatic Infants with SMA

January 25, 2022

The U.S. Food and Drug Administration has granted Priority Review to Genentech and PTC Therapeutics for a supplemental new drug application for Evrysdi to expand the indication to include pre-symptomatic infants under 2 months old with spinal muscular atrophy. If approved, Evrysdi would be the first medicine administered at-home for pre-symptomatic babies with SMA.

Photo: Stuart Peltz, CEO of PTC Therapeutics

“The results demonstrating that almost all of the pre-symptomatic infants achieved motor milestones comparable to healthy infants is tremendous,” said Stuart Peltz, CEO of PTC Therapeutics. “The granting of the Priority Review for Evrysdi recognizes this and the significant need to treat babies with SMA as early as possible.”

Included in the sNDA submission was interim data from the RAINBOWFISH study, which showed 80 percent of pre-symptomatic infants with SMA treated with Evrysdi for at least 12 months achieved motor milestones such as sitting without support, rolling, crawling, standing unaided, and walking independently.

Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and when untreated is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to progressive muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. It is designed to distribute evenly to all parts of the body and 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, which is based on PTC’s splicing platform, 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. The FDA recently approved Evrysdi for the treatment of spinal muscular atrophy for adults and children 2 months and older. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group, and marketed in rest of world by Roche.

Author: Rare Daily Staff

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