Summary:
The IONS and BIIB AdComm meeting for Tofersen is scheduled for this Wednesday, March 22!
Briefing docs were released today (March 20)
There are even more exciting AdComms coming in the next few months
IONS & BIIB AdComm
An AdComm meeting is scheduled for March 22, 2023 (Wednesday) for IONS & BIIB's asset Tofersen. Tofersen could be approved on April 25, 2023 (the current PDUFA date) to treat ALS. The briefing documents for this meeting were released today (March 20, 2023)!
The the FDA briefing documents and the BIIB briefing documents.
The Tofersen AdComm meeting and vote will be very interesting to see because of the mixed results from the previous clinical trials. The Phase 3 VALOR study reported data on October 17, 2021 but the primary endpoint did not reach statistical significance. After this negative data, eligibility for the early access program was expanded to include people with SOD1-ALS. While the primary endpoint was not met for patients with SOD1-ALS, there were signs of reduced disease progression across secondary and exploratory endpoints.
Clinical benefits were determined and in July 2022 the FDA accepted the NDA and granted priority review for Tofersen. In the briefing documents, the FDA did raise concerns, primarily statistical in nature. The FDA called the VALOR study a "greatly underpowered" study because of the lower than expected decline in plasma NfL concentration in the placebo and treatment groups. There also was little evidence on the effect of tofersen on clinical outcome. Even with these concerns, the FDA still may look for a full approval in April during the PDUFA date.
It will be interesting to see the AdComm decision on Wednesday based on this VALOR data and how IONS and BIIB stocks will move. The two questions the AdComm will vote on are:
Is the available evidence sufficient that reduction in plasma NfL concentration in tofersen-treated patients is reasonably likely to predict clinical benefit of tofersen for treatment of patients w/ SOD1-ALS?
Does the clinical data from placebo-controlled study and available long-term extension study results provide convincing evidence of the effectiveness of tofersen in the treatment of patients with SOD1-ALS?
We look forward to the AdComm meeting on Wednesday to see what the committee has determined!
Other AdComms Coming
See Table 1 for other upcoming AdComm meeting.
Table 1. Upcoming AdComms
PDUFAs Coming in March and April
See Table 2 for PDUFAs coming in the next month!
Table 2. Upcoming PDUFAs
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