FDA releases five new guidance documents to advance the development of novel treatments for neurological conditions

Today, the FDA has issued five guidance documents focused on advancing treatments for neurological disorders that aren’t adequately addressed by available therapies. These guidance documents provide details on how researchers can best approach drug development for certain neurological conditions – Duchenne muscular dystrophy (DMD) and closely related conditions, migraine, epilepsy, Alzheimer's disease and amyotrophic lateral sclerosis (ALS). These documents provide our current thinking and sound regulatory and scientific advice for product developers so that safe and effective treatments can ultimately be made available to patients. 

 

The newly released guidance documents include:

• Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry 
• Drugs for Treatment of Partial Onset Seizures: Full Extrapolation of Efficacy from Adults to Pediatric Patients 4 Years of Age and Older Guidance for Industry
• Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry
• Early Alzheimer’s Disease: Developing Drugs for Treatment Guidance for Industry 
• Migraine: Developing Drugs for Acute Treatment Guidance for Industry

 

The guidance documents we have issued today are an important step in facilitating efficient development of treatments for patients with serious neurological conditions. But they are also just one part of our ongoing efforts to modernize the drug review process and foster beneficial new innovation. These changes, both outward facing and internal, will improve our ability to engage with sponsors, patients, and researchers, and adapt quickly to an environment where the science is changing at a breathtaking speed across many disease areas.    

 

You may find additional information in the Statement from FDA Commissioner Scott Gottlieb, M.D. on advancing the development of novel treatments for neurological conditions; part of broader effort on modernizing FDA’s new drug review programs.