The approval in EU is based on interim data from the Phase III trial in children and adolescents with PNH. Credit: allinonemovie / Pixabay.

The EU has granted expanded approval to division Alexion鈥檚听Ultomiris (ravulizumab) for use in children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).

A long-acting C5 complement inhibitor, Ultomiris provides quick, complete and long-lasting complement inhibition.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile 鈥� free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

A very rare and severe blood disorder, PNH is characterised by the damage of red blood cells that can lead to blood clots or thrombosis. This causes organ damage and potentially premature death.

The approved dosage of Ultomiris is every four or eight weeks, on administering a loading dose.

Furthermore, the drug is intended for use in children with a bodyweight of 10kg or above.

In July, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) had

The latest approval is based on interim data from the Phase III clinical trial in children and adolescents with PNH that showed the safety and efficacy of Ultomiris听in these subjects.

In addition, the drug was shown to be effective in attaining complete C5 complement inhibition through 26 weeks in subjects aged up to 18 years in the trial.

No drug-related severe adverse events were observed in the听trial. Ultomiris鈥� efficacy and safety profile in children and adolescents were in line with those observed in adults.

Alexion CEO Marc Dunoyer said: 鈥淭his approval of Ultomiris reflects our ongoing commitment to delivering new treatments that can make a meaningful difference in patients鈥� lives.

鈥淯ltomiris has become the standard of care for the treatment of adults with PNH and we will make it听available to this younger patient population as soon as possible.鈥�

With established safety and efficacy profile, Ultomiris听requires low dosing frequency versus听Soliris听(eculizumab), which is the present standard of care in the EU to treat children and adolescents with PNH.

Ultomiris听obtained approval in the EU to treat adults with PNH in 2019 and is also approved to treat atypical haemolytic uraemic syndrome (aHUS) in adults and children.

Last month, Alexion announced plans to听听for the treatment of amyotrophic lateral sclerosis.