Catalyst overview
Research summary
This catalyst tracks a Approved update for Nusinersen (SPINRAZA) in Spinal muscular atrophy . The expected timing is based on the latest available company guidance and may change.
DEVOTE study. 9/4/24- Positive topline data from the pivotal cohort (Part B) of Ph2/3. 1/23/25- FDA & EMA accepted application for higher dose regimen. 6/27/25- Reported new data. 9/24/25- CRL issued for the high dose regimen sNDA. Plans to resubmit based upon readily available information. 01/12/26:- EC approved high-dose SPINRAZA regimen; FDA decision on high-dose expected by April 3, 2026. 03/05/26:- Present DEVOTE/ONWARD high-dose data at MDA Mar 8-11, 2026. FDA decision expected April 3, 2026. 03/30/26:- FDA approved high-dose SPINRAZA for SMA; U.S. availability in coming weeks.
Verification
Confirm timing and program details against the company announcement or filing.