SOUTH SAN FRANCISCO (dpa-AFX) - Genentech, the U.S. unit of Swiss drug maker Roche Group (RHHBY), reported Monday positive 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy or SMA.
The company noted that the 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones.
SMA is a severe, progressive neuromuscular disease that can be fatal, and is the major genetic cause of infant mortality.
In the trial, the exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% of infants were able to sit without support for at least 5 seconds, 65 percent had maintained upright head control, 29% could turn themselves over and 30% were able to stand either supporting weight or with support.
The most serious adverse event that occurred in 24% of infants was pneumonia.
Levi Garraway, chief medical officer and head of Global Product Development, said, 'We are highly encouraged by the results we are seeing in the second year of treatment with Evrysdi. ...we look forward to continued assessments of both survival and motor function during long-term follow up for this first-of-its-kind treatment.'
The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.
Evrysdi is being studied in more than 450 people as part of a broad and robust clinical trial program in SMA.
The U.S. Food and Drug Administration approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older.
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