FDA grants Breakthrough Therapy designation to fosigotifator for vanishing white matter disease

Calico/Fosigotifator (ABBV-CLS-7262)

The FDA granted Breakthrough Therapy Designation to fosigotifator, Calico's eIF2B activator, for vanishing white matter disease, a progressive neurological condition with no approved treatment. The drug is in Phase 1b/2 trials in adults, children and infants.

• eIF2B activator targets the integrated stress response; preclinical work showed it increased activity of VWM-associated eIF2B mutations.
• Vanishing white matter is an ultra-rare, progressive condition affecting brain white matter with onset typically ages 2-6.
• First eIF2B activator ever given to humans with VWM; trial evaluates safety, tolerability, pharmacokinetics and efficacy.