FDA Grants Breakthrough Therapy Designation to Calico's Fosigotifator
Calico Life Sciences LLC has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to fosigotifator for the treatment of Vanishing White Matter disease, a rare and progressive neurological disorder with no approved therapies.
Fosigotifator is an investigational eIF2B activator currently being evaluated in a Phase 1b/2 clinical trial involving adult, paediatric and infant patients with VWM disease.
The study is assessing the therapy’s safety, tolerability, pharmacokinetics and exploratory efficacy.
The FDA awards Breakthrough Therapy Designation to investigational medicines that show the potential to offer substantial improvement over existing treatment options for serious or life-threatening conditions.
The designation follows fosigotifator’s earlier selection for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) programme.
VWM disease is caused by mutations affecting the eIF2B enzyme, resulting in damage to the brain’s white matter and progressive neurological decline.
Preclinical studies suggest fosigotifator may restore eIF2B activity, reduce disease-related stress responses and improve neurological function.
The ongoing Phase 1b/2 study (NCT05757141) is the first clinical trial to evaluate an eIF2B activator in patients with VWM disease. Fosigotifator remains an investigational therapy and has not been approved by any regulatory authority.
