FDA Amyotrophic Lateral Sclerosis Developing Drugs for Treatment Guidance for Industry

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Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment 1

Guidance for Industry This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA office responsible for this guidance as listed on the title page. I. INTRODUCTION The purpose of this guidance is to assist sponsors in the clinical development of drugs and 2

biological products 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 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 3

Clinical Evidence of Effectiveness for Human Drug and Biological Products (May 1998) and 1

This guidance has been prepared by the Division of Neurology Products in the Center for Drug Evaluation and Research in cooperation with the Center for Biologics Evaluation and Research at the Food and Drug Administration. 2

For the purposes of this guidance, all references to drugs include both human drugs and biological products unless otherwise specified. 3

We update guidances periodically. To make sure you have the most recent version of a guidance, check the FDA guidance web page at https://www.fda.gov/regulatory-information/search-fda-guidance-documents.

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