Kizfizo®

About Orphelia Pharma

Orphelia Pharma is a mission-driven company focused on addressing the unmet medical needs of children with rare diseases by developing pediatric-adapted formulations. Founded in 2015 (from TargeOn, originally created in 2007), the company has successfully brought Kigabeq® to market in the EU and advanced Kizfizo® to marketing authorization review. Its strategic acquisition by Orphalan in late 2025 positions it to leverage greater global commercial capabilities while continuing its core mission of improving care for pediatric patients with rare neurological and oncological conditions.

Therapeutic Areas