Rare Daily Staff

The European Union has expanded the approved use of Ultomiris to include children and adolescents with paroxysmal nocturnal hemoglobinuria, an ultra-rare and severe blood disorder.

The expanded use of Ultomiris follows the U.S. Food and Drug Administration’s similar expansion in June. Ultomiris has already been approved in the United States, European Union, and Japan for the treatment of adults with PNH. It is also approved in the United States and Japan for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy in adult and pediatric (one month of age and older) patients, as well as in the European Union for the treatment of adults and children with a body weight of at least 10 kg with aHUS.

Paroxysmal nocturnal hemoglobinuria (PNH) is a serious ultra-rare blood disorder that is characterized by the destruction of red blood cells, which is also referred to as hemolysis. PNH occurs when the complement system—a part of the body’s immune system—over-responds, leading the body to attack its own red blood cells. PNH often goes unrecognized, with delays in diagnosis from one to more than five years. Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine, and anemia. The most devastating consequence of chronic hemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death. The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.

Ultomiris is a long-acting C5 complement inhibitor that offers immediate, complete, and sustained complement inhibition, and is now the first and only FDA-approved medicine for children and adolescents with PNH. It works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose.

The approval by the European Commission was based on interim results from the phase 3 clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients.

Ultomiris has an established safety and efficacy profile, offering reduced dosing frequency compared to Soliris, which is the current standard of care in the European Union for the treatment of children and adolescents with PNH. Ultomiris is administered every four or eight weeks (depending on body weight), following a loading dose. The approval follows the recommendation of the Committee for Medicinal Products for Human Use of the European Medicines Agency in July 2021.

“Ultomiris has become the standard of care for the treatment of adults with PNH and we will make it available to this younger patient population as soon as possible,” said Marc Dunoyer, Chief Executive Officer, Alexion.

“Ultomiris has become the standard of care for the treatment of adults with PNH and we will make it available to this younger patient population as soon as possible,” said Marc Dunoyer, CEO of Alexion.

Photo: Marc Dunoyer, CEO of Alexion

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