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Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry

This guidance document is being distributed for comment purposes only.

Comments and suggestions regarding this draft document should be submitted within 60 days of publication in the Federal Register of the notice announcing the availability of the draft guidance.

INTRODUCTION

The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of amyotrophic lateral sclerosis (ALS). 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. This guidance addresses the clinical development of drugs intended to treat the main neuromuscular aspects of ALS (i.e., muscle weakness and its direct consequences, including shortened survival). This draft guidance is intended to serve as a focus for continued discussions among the Division of Neurology Products, pharmaceutical sponsors, the academic community, and the public. 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.

Click here to download the full FDA draft guidance document.