EU regulator backs AstraZeneca drug for rare blood disorder in children

AstraZeneca said on Monday the European Medicines Agency (EMA) recommended approving a medicine from its U.S.-based Alexion unit for treating a rare, deadly blood disorder in children and adolescents aged up to 18 years.

The drugmaker recently completed its $39-billion acquisition of Alexion, which it had agreed to buy as a bet on rare-disease immunology and to boost its business that includes a fast-growing cancer medicines unit and a major COVID-19 vaccine.

AstraZeneca said the drug Ultomiris had been given a positive opinion for paroxysmal nocturnal haemoglobinuria (PNH) by the human medicines committee of the EMA, and it follows a U.S. approval in June.

The EMA endorsement is to include children with a body weight of 10 kg or above, and those patients who are clinically stable after having been treated with another Alexion drug, Soliris, for at least the past six months.

“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,” said Austin Kulasekararaj, MD at King’s College Hospital in London.

Ultomiris is an improved version of Alexion’s best-selling drug Soliris, which is used against a range of rare immune-disorders including PNH where the body’s immune system attacks its own red blood cells causing anaemia and blood clots.