– Evrysdi is the first and only at-home administered treatment for patients living with SMA
– Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date
PTC Therapeutics, Inc. announced on May 31st 2022 that the US Food and Drug Administration (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. 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.Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics.
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.
About Spinal Muscular Atrophy (SMA)
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.
About Evrysdi® (risdiplam)
Evrysdi 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. Evrysdi is designed to distribute evenly to all parts of the body, including the central nervous system (CNS), and it is administered daily at home in liquid form by mouth or feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the FDA in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society, as well as the Society for Medicines Research Award for Drug Discovery. Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group, and marketed in rest of world by Roche.