Replies to post #391471 on Anavex Life Sciences Corp (AVXL)

[abew4me]

So it's okay for the FDA to approve Biogen's adu-crap because it had two large studies - and therefore it checks all of the "standardized" boxes. Never mind that it causes brain bleeds and death.

Hypocrisy at its highest!

[sokol]

“Accelerated approval recognizes that physicians and patients are generally willing to accept greater risks or side effects from products that treat life-threatening and severely-debilitating illnesses, than they would accept from products that treat less serious illnesses.”

https://undark.org/2018/09/10/fda-drugs-accelerated-approval/

Accelerated approval has been used in cancer trails, ALS trials, and in the case of Alzheimer’s when measurement of a drug’s effect is lengthy, expensive and there is an unmet need. Efficacy is proven later in a confirmatory trial.

If Blarcamesine is reasonably safe and if Anavex can show through a surrogate endpoint that Blarcamesine is “reasonably likely to predict clinical benefit”, it may gain accelerated approval. Efficacy may then be established later in a confirmatory trial. Approval of Aduhelm was unexpected. Scientist and doctors were saying it should not be approved because of efficacy and safety issues. Nevertheless, the FDA approved Aduhelm.

“In announcing its (Aduhelm’s) approval .., the F.D.A. acknowledged there was not sufficient evidence that the drug would help patients. Instead, it said it was greenlighting Aduhelm under a program called “accelerated approval,” which allows the authorization of drugs without persuasive proof of benefit if they are for serious diseases with few treatment options and if the drug affects part of the disease’s biology (known as a biomarker) in a way that is “reasonably likely to predict clinical benefit.””

https://www.nytimes.com/2021/07/19/health/alzheimers-drug-aduhelm-fda.html

[Anshu2]

FDA rules may not seem common sense to you and many, but they are governed by laws. And, IMO, thats the right approach to approving drugs.

SRPT's fans (including 150 congressmen, who wrote a letter to FDA, and Marco Rubio who gave a speech in Senate) kept on begging FDA to approve SRPT's Eteplirsen. During those times, it seemed like a no-brainer to approve Eteplirsen (it was safe, and kids were dying/losing muscles). BUT --- FDA was very very clear. It was going to do an AA ONLY if the drug studies showed " substantial evidence that the drug expressed/impacted the surrogate marker (dystrophin expression". SRPT's drug was almost almost rejected, but JW put her foot down and went against AdCom as well as staff, and unilaterally (pretty-much) decided to approve Etep.

Note that the bar of substantial evidence is NOT LOWERED for AA (compared to full-approval). The only differences between AA and full-approval are:

1. Use a surrogate market (or intermediate endpoint) as the endpoint to evaluate.
2. Then, do a confirmatory trial to evaluate the clinical/final endpoint.

Safety doesn't really play a critical role. Efficacy does. If a drug is efficacious, then FDA will approve as long as the safety risk is not high. NOT THE OTHER WAY ROUND, i.e., FDA will not approve a perfectly safe drug with non-so-solid evidence of efficacy. The latter may seem against common sense, but thats how it is (IMO, rightly so).

[oldandintheway]

Abe, common sense departed the medical profession a long time ago...