Roche (RHHBY) said that new four year data for its Evrysdi reinforced long-term efficacy and safety profile in some of the most severely affected people with types 2 and 3 spinal muscular atrophy.
Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease over the 48 month period.
Participants also reported continuous improvement or stabilization when independently performing activities of daily living such as eating, drinking and picking up and moving objects.
More than 8,500 people—from newborns to the over 60s—have been treated with Evrysdi , which is now approved in more than 90 countries worldwide.
In addition, Genentech said it leads its clinical development as part of a collaboration with the SMA Foundation and PTC Therapeutics. Genentech is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Phase II/III trial MANATEE for the treatment of SMA.
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
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