EU regulator backs AstraZeneca drug for rare blood disorder in children

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[July 26, 2021]  (Reuters) -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.

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"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.

(Reporting by Pushkala Aripaka in Bengaluru; Editing by Ramakrishnan M and Uttaresh.V)

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