Roche touts 2-year efficacy data for SMA therapy Evrysdi; NIH funds new network to study flu and viruses with ‘pandemic potential’
Two years after therapy, 61% of babies with spinal muscular atrophy Type 1 treated with Roche’s Evrysdi (risdiplam) were able to sit up without support for at least five seconds compared with just 29% of patients after a year, the drugmaker said Thursday.
As part of the FIREFISH-2 Phase III study, researchers tested Evrysdi’s ability to help 41 infant patients sit up without support at 5 and 30 seconds. Data showed that 44% of patients were able to sit without support for 30 seconds at the two-year check-in compared with 17% after one year.
Ninety-three percent of infants were alive after 24 months of treatment, Roche said, and 83% percent of patients (34/41) were alive and free from permanent ventilation at that mark. Without treatment, the median age of death or permanent ventilation is 13.5 months, Roche said. No new deaths were reported between months 12 and 24.
On the study’s secondary endpoints, Evrysdi also maintained patients’ motor function at two, with 92% of patients being able to feed orally and 95% maintaining the ability to swallow, Roche said. — Kyle Blankenship
NIH funds a new network to study flu and viruses with ‘pandemic potential’
After funding ran out on the NIAID’s last flu research and surveillance program, the agency has established and funded a new network to track the flu, Covid-19, and other viruses with pandemic potential.
The new program, called the Centers of Excellence for Influenza Research and Response (CEIRR), includes three of the research institutions involved in the last program, with two additions. NIAID contracts will support the network for the next seven years, with the first year of contracts totaling $24 million.
The goal is to study and surveil the transmission of influenza, including from animals to humans, to better understand how viruses evolve and adapt. Although the primary focus is on the flu, the network will also study SARS-CoV-2, and other emerging viruses of concern.
CEIRR replaces the former Centers of Excellence for Influenza Research and Surveillance (CEIRS) program, which was funded by contracts that expired at the end of last month.
The institutions awarded contracts this time around include: the Icahn School of Medicine at Mount Sinai for about $6 million; the University of Pennsylvania for nearly $7 million; St. Jude Children’s Research Hospital for nearly $9 million; Emory University for just over $1 million; and the University of Georgia Research Foundation for exactly $1 million. — Nicole DeFeudis