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On 30 March 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Ultomiris. The marketing authorisation holder for this medicinal product is Alexion Europe SAS.

The CHMP recommended the addition of a new pharmaceutical form (solution for injection in a cartridge) associated with a new strength (245 mg) to be administered subcutaneously via an on-body injector.

The full indications for the new formulation of Ultomiris will be as follows:

  • the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH):
    • in patients with haemolysis with clinical symptom(s) indicative of high disease activity.
    • in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.
  • the treatment of adult patients with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

Key facts

Agency product number
International non-proprietary name (INN) or common name
  • ravulizumab
Active substance
  • ravulizumab
Therapeutic area
Hemoglobinuria, Paroxysmal
Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Date opinion adopted
Company name
Alexion Europe SAS
Application type

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