Anavex Life Sciences Corp (AVXL)

[Anshu2]

FDA (full) approves a drug only if there is “substantial evidence” for efficacy.

Now what is efficacy? Best definition is “clinically meaningful benefit”.

FDA has explicitly said that they don’t mind if the benefit is “small” (but clinically meaningful) — but the evidence for it still needs to be “substantial”.

Safety is not really a stringent requirement (but efficacy is) —- but the benefit/risk ratio (judgement call) needs to be favorable.

In our context of AVXL, the benefit needs to be statistically significant AND high enough (since it’s only a single trial). Else, forget about approval. Don’t expect “unmet need” to soften FDA’s stance — you have no idea how hardened FDA was wrt SRPT even though the Congress begged for it (but yes, eventually approved after a lot of in-fighting and resignations).