conditional approvals in the CNS space and came across a few examples:
Yes, the EMA has done conditional approval for SMA and DMD but these differ greatly from AD. SMA US plus EU has about 1000 cases/year (drug approved up to age 2 or 21kg) so only up to 2000 cases eligible for treatment, So obviously a 1000 patient trial could never be done. Exondys is approved only for those with exon51 mutation (13% of DMD) and only in ambulatory patients (excludes most > age 10). Rett could possibly have used this path is benefit was overwhelming (note trofinetide never got approval in EU though they did submit an MAA - likely with their phase 3 plus experience over last 2 years in US). But the drug failed.
AD has 20MM patients in US plus EU, Japan, AU of which 1/4-1/3 are MCI or mil. The only reason to run a small (169 per cohort vs 375 per cohort proposed by ANVS, 800+ per cohort for MAbs), short trial when other drugs did properly powered trials was to save money - not because patients are unavailable or trials are difficult. Additionally with mismatched baseline on ADAS-Cog and MMSE (large issue when ADAS-cog endpoint has nonlinear progression) and high dropout, data is suggestive but weak. I think this lack of data would have been a prominent LOQ point. Missling will try to finesse the absence of data by sending back OLE data (huge dropout) and biomarker/MRI (moderate dropout- no hippocampal volume) data and promising to do a confirmatory trial if approved (though couldn't be bothered to start one before submission). CHMP currently reviewing the responses to prepare letter of outstanding issues. We'll know soon, probably after Schizo readout
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