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July 1, 2020

Alexion drug wins approval in Europe to treat rare blood disease

PHOTO | Contributed Alexion, AstraZeneca Rare Disease's research facility at 100 College St. in New Haven.
By Natalie Missakian

Alexion Pharmaceuticals' follow-up drug to its blockbuster Soliris has been cleared by regulators in the European Union to treat patients with a rare life-threatening blood disease.

The approval by the European Commission, announced Monday, clears the way for Alexion to market the drug to treat adults and children with atypical hemolytic uremic syndrome (aHUS). 

The treatment prevents the body from attacking itself by inhibiting C5, an immune system protein that is overactivated in people with aHUS. 

The disease causes blood clots and blood vessel damage, and prognosis can be poor, with many patients developing end-stage kidney disease or dying within a year of diagnosis. 

Results from a clinical trial showed both adults and children with aHUS had complete C5 inhibition after the first dose of Ultomiris, which lasted for up to eight weeks.

“At Alexion, our goal is to continue to improve the lives of people and families affected by aHUS and other serious rare diseases,” said John Orloff, MD, Alexion’s head of research and development. “Today’s marketing authorization marks an important step in our efforts to establish Ultomiris as the new standard of care for the aHUS patient community.”

The EU approval was for patients who have never been treated with a complement inhibitor or who have received and responded to Soliris for at least three months.

Ultomiris was approved to treat aHUS in the U.S. last October. Its initial FDA approval in Dec. 2018 was for another rare blood disease, paroxysmal nocturnal hemoglobinuria (PNH). It is also approved to treat PNH in Europe and Japan.

Alexion is still awaiting regulatory approval to market Ultomiris to aHUS patients in Japan. 

Contact Natalie Missakian at news@newhavenbiz.com.

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