Anavex Life Sciences Corp (AVXL)

[XenaLives]

Link to guidance doc on ALS just released
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https://www.fda.gov/media/130964/download

IMO this is part of the transition to precision medicine.

Better biomarkers, shorter trials.

I. INTRODUCTION
The purpose of this guidance is to assist sponsors in the clinical development of drugs and
biological products for the treatment of amyotrophic lateral sclerosis (ALS).
2 Specifically, this
guidance addresses the Food and Drug Administration’s (FDA’s) current thinking regarding the
clinical development program and clinical trial designs for drugs to support an indication for the
treatment of ALS.
ALS is a progressive neurodegenerative disease that primarily affects motor neurons in the
cerebral motor cortex, brainstem, and spinal cord, leading to loss of voluntary movement and the
development of difficulty in swallowing, speaking, and breathing and shortened life expectancy.
This guidance addresses the clinical development of drugs intended to treat the main motor
aspects of ALS (i.e., muscle weakness and its direct consequences, including shortened survival).
This guidance does not address in detail the development of drugs to treat other symptoms that
may arise in ALS, such as muscle cramps, spasticity, sialorrhea, pseudobulbar affect, and others.
This guidance focuses on specific clinical drug development and trial design issues that are
unique to the study of ALS. General issues of concern in drug development, such as the quantity
of effectiveness evidence needed to support approval for serious and life-threatening diseases or
approaches to adaptive study design, are discussed in the guidance for industry Providing
Clinical Evidence of Effectiveness for Human Drug and Biological Products (May 1998)3 and
the draft guidance for industry Adaptive Designs for Clinical Trials for Drugs and Biologics (September 2018),
4 respectively. This guidance also does not contain discussion of the
general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH
guidances for industry E9 Statistical Principles for Clinical Trials (September 1998) and E10
Choice of Control Group and Related Issues in Clinical Trials (May 2001), respectively.
In general, FDA’s guidance documents do not establish legally enforceable responsibilities.
Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only
as recommendations, unless specific regulatory or statutory requirements are cited. The use of
the word should in Agency guidances means that something is suggested or recommended, but
not required.