Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –

– Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –

BOSTON, MA, USA I June 7, 2021 IAlexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 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 can take months, and sometimes years, to receive a correct diagnosis for PNH — a chronic, progressive and potentially life-threatening rare disease – which can be an overwhelming experience for children and their families,” said Satheesh Chonat, M.D., principal investigator for the pediatric clinical trial and pediatric hematologist and oncologist at the Aflac Cancer & Blood Disorders Center at Children’s Healthcare of Atlanta, as well as assistant professor of pediatrics at the Emory University School of Medicine. “Managing the disease can be extremely burdensome for these children and their families, who often miss school and work for infusions, blood transfusions, and medical appointments. It’s exciting to finally have an approved medicine for these patients who are diagnosed as children.”

Since its initial approval in 2018, ULTOMIRIS has quickly become the standard of care in the U.S. for the treatment of adults with PNH. PNH is a complement-mediated disease, which means the symptoms and complications are caused by a lack of regulation, or control, of the complement system, an essential part of the immune system. ULTOMIRIS is designed to target the part of the complement system at the site of disease activity (terminal complement), while preserving function of other parts of the immune system to be able to fight common pathogens and infections. ULTOMIRIS reduces red blood cell destruction in the blood vessels, also known as intravascular hemolysis, and thrombosis (blood clot) risk by providing immediate, complete, and sustained terminal complement inhibition.

“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, Chief Executive Officer and Executive Director of the Aplastic Anemia and Myelodysplastic Syndrome International Foundation (AAMDSIF). “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.”

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. The efficacy and safety of ULTOMIRIS in children and adolescents is consistent with the established profile of ULTOMIRIS in clinical studies involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting. The results of the pediatric study continue to demonstrate Alexion’s commitment to treating complement-mediated diseases, which spans more than 17 clinical trials – including the largest pediatric and adult trials in PNH completed to date – and over 60,000 patient-years of trial and real-world data, across more than 50 countries. Data from the interim analysis will be presented as an e-poster during the European Hematology Association 2021 Virtual Congress and will be made available on the congress website on June 11, 2021 at 9:00 a.m. CEST (3:00 a.m. EDT).

“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, M.D., Executive Vice President and Head of Research and Development at Alexion. “We are inspired by the bravery and resilience of the children and adolescents, as well as their families, who participated in the study, and we are grateful for their commitment — as well as that of the trial investigators — to advancing the understanding of PNH and disease management in younger people. We also appreciate the sense of urgency shown by regulators in prioritizing reviewing and approving the first treatment in the U.S. for children and adolescents with PNH.”

Alexion plans to make ULTOMIRIS available to pediatric patients in the U.S. immediately. A regulatory filing for ULTOMIRIS in pediatric patients with PNH is under review in the European Union (EU).

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a serious ultra-rare blood disorder with devastating consequences. It 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® (ravulizumab-cwvz) is the first and only long-acting C5 complement inhibitor. The medication 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. ULTOMIRIS is approved in the United States (U.S.) for the treatment of adults and children (one month of age and older) with paroxysmal nocturnal hemoglobinuria (PNH), as well as in the European Union (EU) and Japan as a treatment for adults with PNH. It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit




ULTOMIRIS is a prescription medicine used to treat:

  • adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at:

SOURCE: Alexion Pharmaceuticals

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