Anavex Life Sciences Corp (AVXL)

[Steady_T]

The next AD trial may not be a P4 due to AA. AD may be a straight up approval. The FDA can grant Priority Review status upon NDA submission.

FDA decides on the review designation for every application. However, an applicant may expressly request priority review as described in the Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics. It does not affect the length of the clinical trial period. FDA informs the applicant of a Priority Review designation within 60 days of the receipt of the original BLA, NDA, or efficacy supplement. Designation of a drug as “Priority” does not alter the scientific/medical standard for approval or the quality of evidence necessary.

Rett may be an AA or a straight up approval. To obtain an AA a company must still submit the same data package that is required for a straight up approval. Since Rett has rolling submission authority via Fast Track designation there shouldn't be much difference in the approval time frame for AA and a straight up approval.

Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track

I think Missling is using the term Accelerated approval to mean rapid approval rather than the specific AA process.

So far I don't see any designated surrogates in any trial. There are a number of measurements and lab results that could possibly be used as surrogates but I haven't seen them validated in any Rett trial. For the AD trial Anavex is measuring a variety of CSF parameters. There are no Pet scans.

CSF assessment [ Time Frame: 48 weeks ]
Changes in CSF parameters (Abeta40, Abeta42, T-tau, P-tau, NFL, YKL-40, neurogranin, BACE1 concentration) characteristic for AD pathophysiology from baseline and compared to placebo at

+48 weekstreatment differences within subgroups will be performed

Whether the FDA would accept those measurements are surrogates is an unknown.