logo
  

PTC Therapeutics : FDA Approves Label Extension For Evrysdi For Infants With Spinal Muscular Atrophy

PTC Therapeutics Inc. (PTCT) said Tuesday that the U.S. Food and Drug Administration has approved a label extension for Evrysdi (risdiplam) to include infants under 2 months old with spinal muscular atrophy or SMA.

The approval was based on interim efficacy and safety data from the RAINBOWFISH study in newborns. The data 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.

Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal.

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, and it is administered daily at home in liquid form by mouth or feeding tube.

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.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
The U.S. Food and Drug Administration said it now intends to review only a small subset of new emergency use authorization or EUA requests for diagnostic tests. As per an updated COVID-19 test policy with a view to ensure continued access to tests, the regulator further urged developers of all test types seeking marketing authorization to pursue traditional premarket review for most test types. Netflix, Inc. said it is adding a new feature to offer a more personalized mobile games experience on the platform. In a statement, Sophia Yang, Product Manager, Mobile Games, said the company is rolling out the ability to create game handles, with which members will be able to create a unique public username. This can be used across all Netflix games. General Motors Co. has delayed its plan to require employees to return to office many days a week until next year, reports said citing an internal memo signed by CEO Mary Barra. Last week, the company had announced that corporate workers would be required to return to respective offices at least three days a week, beginning later this year, as the COVID-19 pandemic eases.
RELATED NEWS
Follow RTT