Anavex Life Sciences Corp (AVXL)

[Doc328]

Drugs can be approved by the FDA without trials in the US though this is very rare. I am aware of only one neurology drug that was approved based on 2 trials outside the US (Edaravone for ALS) though I am sure there were others.


The FDA published guidance to industry. Here's a relevant guidance. Here's another interesting link that has less bureau-speak

Despite claims by some, practically all countries conducting clinical trials must follow similar guidelines. The ICH established rules for clinical studies and established 'Good Clinical Practice' quality standards. This has allowed international studies across continents to be seamless and lead to results that will be judged in a similar manner by the regulatory agencies. All investigators are trained in GCP/ICH (several hours every two years I never get back).

Studies in the US are more expensive than many countries (but about the same as UK and Germany). Although it is possible to get approved by the FDA without a US site, most pharma sponsors still want to have US sites. There are many experienced investigators in the US with proven track records of quality performance and good recruitment. Additionally, and this may become even more important going forward, the FDA is very concerned about under-representation of minorities. The US is 40% minorities and the FDA wants studies to be inclusive. Obviously, EU, AU, China are under-represented in populations that resemble the African American and Hispanic American populations. Some BP have run minority studies in the US (I have done for MS) after a drug is approved with negligible numbers of non-white subjects. Here's anotehr guidance

IMO, there is little chance of approval based on one 450 patient study so Anavex will do a phase 3 including US sites if results are good in the smaller study.