Ultomiris recommended for approval in the EU by CHMP for children and adolescents with paroxysmal nocturnal haemoglobinuria

Alexion’s Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on interim results from the Phase III clinical trial in children and adolescents with PNH, which were recently presented during the European Hematology Association 2021 Virtual Congress.

This trial demonstrated that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of children and adolescents up to 18 years of age with PNH. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.¹

The efficacy and safety of Ultomiris in children and adolescents is consistent with the established profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.

PNH is an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis (blood clots), which can occur throughout the body, and result in organ damage and potentially premature death.

Austin Kulasekararaj, MD, King’s College Hospital, London, United Kingdom, said: “PNH can have a significant and devastating impact on a child’s quality of life, and it can be overwhelming for families to manage their disease. 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.”

Marc Dunoyer, incoming Chief Executive Officer, Alexion, said: “This recommendation shows that Ultomiris – which has become the standard of care for the treatment of adults with PNH – has the potential to transform the lives of children and adolescents in Europe suffering from this devastating rare disease. As we listen to the patient community and understand the challenges of living with a rare disease, we recognize the importance of continuing to deliver options and formulations that enhance patient care and disease management.”

The CHMP recommended the expanded use of Ultomiris to include children (with a body weight of 10 kg or above) and adolescents with PNH who experience haemolysis with clinical symptom(s) indicative of high disease activity, as well as for individuals who are clinically stable after having been treated with Soliris for at least the past six months.

Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the US Food and Drug Administration approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.