October 18, 2019

The U.S. FDA has approved a new indication for Ultomiris® (ravulizumab-cwvz), manufactured by Alexion Pharmaceuticals. The product is now indicated to inhibit complement-mediated thrombotic microangiopathy (TMA) in adults and pediatric patients at least one month of age who have atypical hemolytic uremic syndrome (aHUS).

Atypical hemolytic uremic syndrome affects approximately one out of every 500,000 people in the US per year. The rare condition is characterized by complement-mediated TMA, or the formation of blood clots in small blood vessels caused by chronic, uncontrolled activation of the complement system. The complement system is a part of the body’s immune system and is responsible for enhancing the body’s ability to remove pathogens and damaged cells and produce an inflammatory response. Patients with TMA experience a low platelet count (thrombocytopenia), hemolytic anemia (low red blood cell counts due to the destruction of the cells), and acute kidney injury.

In clinical studies, 54% of adults and 71% of children achieved complete TMA response after 26 weeks of treatment with Ultomiris. A complete response was defined by normalization of platelet count and lactate dehydrogenase (an enzyme that can be indicative of tissue damage if present in high amounts), as well as improvement in kidney function (defined as a 25% or greater improvement in serum creatinine from baseline). Ultomiris reduced thrombocytopenia in 84% of adults and 93% of children, reduced the destruction of red blood cells in 77% of adults and 86% of children, and improved kidney function in 59% of adults and 79% of children. Data provided for pediatric patients is interim data, as the pediatric study is ongoing.

Recommended dosing under the new indication is based on the patient’s body weight, and has a loading dose range of 600mg to 3,000mg. Two weeks following administration of the loading dose, patients should be started on a maintenance dose, which ranges from 300mg to 3,600mg based on weight. For patients weighing at least 5kg (11 pounds) and less than 20kg (44 pounds), the maintenance dose should be administered once every four weeks. For patients who weigh 20kg or more, dosing frequency is once every eight weeks. Ultomiris is administered via intravenous (IV) infusion.

A black box warning cautions that the use of Ultomiris is associated with life-threatening meningococcal infections and sepsis. These conditions may advance rapidly and be fatal if not recognized and treated early. The black box warning advises prescribers to comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Patients should be immunized with meningococcal vaccines at least two weeks prior to starting Ultomiris unless the risk of delaying treatment outweighs the risk of meningococcal infection. Patients should be monitored for early signs of meningococcal infection even if vaccinated, as the vaccine does not completely eliminate the risk of infection. Due to the risk of meningococcal infections, Ultomiris must be obtained through a Risk Evaluation and Mitigation Strategy (REMS) program.

First FDA-approved in 2018, Ultomiris is also indicated to treat adult patients who have paroxysmal nocturnal hemoglobinuria (PNH).

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