Anavex Life Sciences Corp (AVXL)

[Steady_T]

A few quotes from that link. Taken in total, this certainly looks like likely reasons for the delays in the Rett trial. The FDA is in flux and what Anavex is wanting to do falls in the middle of the transition process.

We intend to apply many of the lessons we have learned in creating and operationalizing OCE to break down traditional silos in the development of treatments for other diseases and conditions.

Does that sound like out Rett trial perhaps?

As part of FDA’s broader innovation initiative, we are encouraging the use of state-of-the-art innovations such as adaptive trials, modeling, and simulations to allow an evaluation of a product’s safety and effectiveness. We welcome early engagement with sponsors to discuss the use of these innovative tools to expedite product development.

The Centers are updating guidance to assist sponsors in incorporating modeling and simulation – and applying these tools, for instance, to optimize product dosing based on individual physiology and genetics. CDER is currently collaborating with scientists to develop natural history models in Parkinson’s, Huntington’s, Alzheimer’s, and muscular dystrophy which may facilitate modeling of some aspects of product design and evaluation.

Our work is primarily focused in two distinct areas: supporting use of surrogate endpoints in individual drug and biological product development programs, including by cataloguing those previously used as well as a process to develop novel surrogate endpoints; and by facilitating a public process to support biomarker qualification as a drug development tool.

We are currently working towards developing and publishing several guidances required by the Cures Act to establish the process, taxonomy, and framework for DDT qualification.

FDA has finalized guidance on the use of RWE for devices, and intends to release guidance on RWE for drugs and biologics.