Rare Disease Report

Frontotemporal Dementia Treatment, AL001, Granted an Orphan Drug Designation

AUGUST 08, 2018
Krista Rossi
This morning, August 8, 2018, the US Food and Drug Administration (FDA) granted an orphan drug designation to Alector’s AL001, a human recombinant monoclonal antibody, for the treatment of all patients with frontotemporal dementia (FTD).
 
“FTD is caused in part by a dysfunctional brain immune system, and AL001 is one of our immuno-neurology drugs designed to repair that immune system to elicit a therapeutic benefit,” said Arnon Rosenthal, PhD, chief executive officer at Alector, in a recent statement “We believe that our immuno-neurology approach could potentially have as much impact on brain disorders as immuno-oncology drugs have had on cancer patients.”

AL001 is a human monoclonal antibody designed to increase the therapeutic brain levels of progranulin, a secreted immune modulatory factor which when mutated leads to frontotemporal dementia.

Currently, there are no approved therapies on the market that are capable of addressing the underlying cause of FTD. As such, the disease continues to pose a significant challenge for families, providers, and society as a whole, at least, according to Alector’s chief medical officer Robert Paul.

 “The granting of orphan drug designation is a significant milestone for the AL001 development program, and we are excited to work with leaders and experts in the research, medical and patient advocate communities to bring AL001 to the patients suffering from this disease,” he said.

A rapidly progressing degenerative syndrome characterized by prominent cognitive dysfunction, behavioral and personality changes, and language deficits, FTD is the second most common early-onset form of dementia after Alzheimer’s disease, afflicting approximately 60,000 Americans.  

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