The US Food and Drug Administration (FDA) has welcomed an experimental therapy, fosigotifator, developed by AbbVie Inc. (NYSE: ABBV) and Calico Life Sciences, into its Rare Disease Therapy Pilot Program. The investigational eIF2B activator is currently undergoing a Phase Ib/II clinical trial for the treatment of vanishing white matter (VWM) disease, a rare and devastating neurological condition. This inclusion provides AbbVie and Calico with a direct line of communication with FDA reviewers to navigate clinical development challenges.
Fosigotifator represents a novel approach to treating VWM by targeting eIF2B, a critical regulator of the integrated stress response (ISR) pathway. Chronic activation of this pathway is known to trigger the degeneration of the brain’s white matter, which is characteristic of VWM. The compound has demonstrated the ability to downregulate ISR and has shown clinical benefits in preclinical animal models.
As stated in a recent press release, fosigotifator is also being explored as a potential treatment for amyotrophic lateral sclerosis (ALS), indicating its broad potential in addressing neurodegenerative diseases.- Flcube.com
