Ionis Reports the US FDA Acceptance of NDA and Granted Priority Review of Tofersen for Superoxide Dismutase 1 Amyotrophic Lateral Sclerosis
Shots:
- The NDA was based on the P-I study, a P-I/II study, P-III (VALOR) & OLE study to evaluate tofersen for SOD1-ALS. The application also granted priority review with an expected PDUFA date of Jan 25, 2023
- As reported in Oct 2021, the 6mos. P-III (VALOR) trial did not meet its 1EPs of change from baseline to 28wk. in ALSFRS-R, reduction in disease progression at multiple 2EPs & exploratory EPs. In 12mos. (VALOR) & OLE study, earlier initiation of tofersen led to robust & sustained reductions in neurofilament, a marker of neurodegeneration & reduced decline across multiple efficacy EPs
- Serious AEs were reported in 36.5% in (VALOR) & OLE, and 17.3% discontinued treatment due to an AEs. Biogen will continue its early access program for tofersen in dozen countries
Ref: PRNewswire | Image: Ionis
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