AstraZeneca (LSE:AZN) PLC (LON:AZN) announced that Alexion's Ultomiris has been recommended for marketing authorisation in the EU for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on interim results from the Phase III clinical trial in children and teens with PNH.
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This trial demonstrated that Ultomiris was effective in treating patients of up to 18 years of age over 26 weeks, while there were no treatment-related severe adverse events and no patients stopped taking the drug.
AstraZeneca said that the efficacy and safety of Ultomiris in children and teens are consistent with what has been demonstrated in adults.
PNH is an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis (blood clots), which can occur throughout the body, and result in organ damage and potentially premature death.
"PNH can have a significant and devastating impact on a child's quality of life, and it can be overwhelming for families to manage their disease,” said Austin Kulasekararaj from the King's College Hospital in London.
“The potential approval of Ultomiris, which offers the efficacy and safety already established with Soliris (eculizumab) and requires fewer treatments each year, would have a meaningful impact for children with PNH and their families."