Shots:

• The EMA has accepted the MAA of tofersen for review to treat SOD1 ALS. Tofersen is also under the US FDA’s review with an expected decision on April 25, 2023
• The MAA was based on the P-III (VALOR) study in 108 patients, the P-III OLE study in 95 patients, the P-I study & a P-I/II study of Tofersen. The 12mos. results from (VALOR) & OLE studies were published in NEJM which showed a greater reduction in concentrations of SOD1 in CSF & neurofilament light chains in plasma over PBO @~28wks. but did not improve clinical EPs & was associated with AEs
• Change in ALSFRS-R score @52wks. in early-start & delayed-start cohorts was −6.0 & −9.5, neurologic SAEs (7%). Tofersen's early access program which was accessible in 34 countries will be maintained by Biogen

Ref: PRNewswire | Image: Ionis