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.

Focuses on developing pediatric-appropriate formulations (e.g., oral liquids) and securing orphan drug designation for treatments targeting rare diseases in children, leveraging a partnership-driven development model.

Competes with large pharma orphan drug units (e.g., Novartis for everolimus) and specialized rare disease companies. Key differentiation is exclusive focus on pediatric-adapted formulations for rare neurological/oncological diseases, addressing a critical unmet need where standard adult formulations are unsuitable.