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GUIDANCE DOCUMENT

Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment February 2018

Draft

Not for implementation. Contains non-binding recommendations.

Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment

Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research

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 This draft guidance is intended to serve as a focus for continued discussions among the Division of Neurology Products, pharmaceutical sponsors, the academic community, and the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.


Submit Comments

Submit comments on this guidance document electronically via docket ID: FDA-2013-S-0610 - Specific Electronic Submissions Intended For FDA's Dockets Management Staff (i.e., Citizen Petitions, Draft Proposed Guidance Documents, Variances, and other administrative record submissions)

If unable to submit comments online, please mail written comments to:

Division of Dockets Management (HFA- 305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

All comments should be identified with the title of the guidance.