EU approval for Ultomiris for children and adolescents with PHN

Following the July recommendation from the Committee for Medicinal Products for Human Use of the European Medicines Agency, Ultomiris (ravulizumab) has now been approved in the European Union for expanded use to include children (with a body weight of 10 kg or above) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare and severe blood disorder characterized by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.

The drug was developed by Alexion, AstraZeneca Rare Disease, the group within UK pharma major AstraZeneca (LSE: AZN) focused on rare diseases that was created following the 2021 $39 billion acquisition of Alexion Pharmaceuticals.

The approval by the European Commission was based on interim results from the Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients.