EVRYSDI (RISDIPLAM) APPROVED BY THE AMERICAN FOOD AND MEDICINE ADMINISTRATION (FDA) TO COVER BABIES UNDER 2 MONTHS!

Evrysdi (Risdiplam) is approved by the US Food and Medicine Administration (FDA) to cover infants younger than 2 months.
Approval is based on provisional efficacy and safety data from the neonate RAINBOWFISH study. Presymptomatic infants treated with Evrysdi achieved significant milestones such as half walking, sitting and standing after 12 months of treatment. All infants survived the 12-month period without permanent ventilation.
Evrysdi aims to treat SMA by increasing and maintaining the production of the SMN protein, which is critical for maintaining healthy motor neurons and movement in the body.
Evrysdi (Risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) directed RNA splicing modifier designed to treat SMA caused by mutations on chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to be distributed evenly to all parts of the body, including the central nervous system (CNS) and is administered daily at home in liquid form by mouth or feeding tube.
Evrysdi is designed to treat SMA by increasing and maintaining the production of survival motor neuron (SMN) protein in the central nervous system and peripheral tissues. SMN protein found in the body; is critical for maintaining healthy motor neurons and movement.
Evrysdi was awarded PRIME by the European Medicines Agency (EMA) in 2018 and an Orphan Drug Designation by the FDA in 2017. Evrysdi is currently approved in 81 countries and the case is under review in 27 more.