Preliminary results from an open-label Phase 3 clinical trial, STR1VE, evaluating Novartis (NYSE:NVS) unit AveXis' gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in patients with spinal muscular atrophy type 1 (SMA Type 1) showed a treatment effect. The data were presented at the MDA Clinical and Scientific Conference in Orlando, FL.
As of September 27, 2018 95.5% (n=21/22) of treated patients were alive and event-free. Median age was 9.5 months with 85.7% (n=6/7) expected to reach at least 10.5 months of age event-free. Per natural history, 50% of babies with SMA Type 1 will not survive or will require permanent ventilation by age 10.5 months.
In the SHINE open-label extension study evaluating Biogen's (NASDAQ:BIIB) SPINRAZA (nusinersen) the median time to death or permanent ventilation was 73.0 weeks (~1.4 years) in SMA patients from the ENDEAR study who continued in SHINE.
As of December 31, 2018 77.3% (n=17/22) of treated patients could hold their head erect for at least three seconds without support while 36.4% (n=8/22) could sit without support for at least 30 seconds.
The company's U.S. marketing application is currently under FDA review with an action date in May. Approvals in Europe and Japan should happen later this year.
Now read: Novartis: Look To Alcon Instead »
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