Roche ’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy (SMA)

Interim data submitted to the FDA show majority of pre-symptomatic babies treated withEvrysdi for at least one year were able to sit, stand and walk within timeframes typical of healthy babies, as well as maintain swallowingEvrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to dateBasel, 25 January 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today 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, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “Extendin g 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) prot...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news