By: IPP Bureau
Last updated : January 27, 2022 2:43 pm
Evrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to date
Roche announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the RAINBOWFISH study, which shows the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking and maintained the ability to swallow following 12 months of treatment.
“Treating very young babies with Evrysdi before SMA symptoms arise may help them to achieve milestones such as standing and walking within timeframes typical of healthy infants,” said Levi Garraway, CMO and Head of Global Product Development, Roche. "Extending treatment access for the youngest members of the SMA community is crucial and we look forward to working with the FDA on this application.”
Evrysdi is designed to treat SMA by increasing and sustaining 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’s existing FDA label is for the treatment of SMA in adults, children and babies two months and older. If approved, Evrysdi would be the first medicine administered at-home for pre-symptomatic babies with SMA.
Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.