Rare Daily Staff

The U.S. Food and Drug Administration approved the expanded use of Alexion Pharmaceuticals’ Ultomiris to include children one month of age and older and adolescents with paroxysmal nocturnal hemoglobinuria.

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, 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.

“PNH can have a profound impact on a child’s development and quality of life. With its established safety and efficacy profile, ULTOMIRIS has the potential to transform the lives of children and adolescents suffering from this devastating rare disease,” said John Orloff, executive vice president and head of research and development at Alexion.

This approval is based on interim phase 3 study results, which showed that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis, which can lead to disabling or potentially fatal blood clots.

Ultomiris has already been approved in the United States, European Union, and Japan for the treatment of adults PNH), as well as in the European Union (EU) and Japan as a treatment for 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.

“This expanded approval is a significant step forward for the PNH community as we work to elevate awareness of this rare disease in children and adolescents and ensure patients, both pediatric and adult, have meaningful treatment options available,” said Janice Frey-Angel, CEO and executive director of the Aplastic Anemia and Myelodysplastic Syndrome International Foundation. “PNH can have significant physical, emotional and/or psychological impacts on families, and we are pleased there is now an approved medicine for the younger members of our community and the families who care for them.”

Alexion plans to make Ultomiris available to pediatric patients in the United States immediately. A regulatory filing for Ultomiris in pediatric patients with PNH is under review in the European Union.

Photo: Janice Frey-Angel, CEO and executive director of the Aplastic Anemia and Myelodysplastic Syndrome International Foundation

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