Ultomiris Approved to Treat Rare Blood Disorder

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Ultomiris Approved to Treat Rare Blood Disorder

December 21, 2018 – The U.S. FDA has approved UltomirisTM (ravulizumab), manufactured by Alexion Pharmaceuticals, to treat adult patients who have paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder.

An acquired disorder, PNH causes blood clots (thrombosis), impaired bone marrow function, and the destruction of red blood cells (leading to hemolytic anemia). The median age of diagnosis is 30-45 years, but the disease can occur in children and adolescents. Up to 30% of patients with PNH have previously had aplastic anemia. Median survival following diagnosis is 10 years.

Ultomiris treats PNH by helping to prevent the destruction of blood cells. In a clinical study, it was found to have similar efficacy to the current standard of treatment, Alexion’s Soliris® (eculizumab).

Unlike Soliris, Ultomiris requires administration only once every eight weeks for maintenance therapy.Soliris must be administered once every two weeks under its PNH indication. Patients treated with Ultomiris can begin the maintenance dose just two weeks after an initial loading dose; those treated with Soliris are titrated up from a starting dose, receiving weekly injections for five weeks before moving to maintenance therapy.

A black box warning advises that life-threatening meningococcal infections/sepsis have occurred in patients treated with Ultomiris. Patients should be immunized against meningococcal infection at least two weeks prior to beginning treatment, and should be monitored for signs of infection. Soliris carries a similar black box warning. Both Soliris and Ultomiris are available only through a Risk Evaluation and Mitigation Strategy (REMS) program.

Ultomiris is expected to launch in 2019 at an estimated average annual cost of $458,000 per patient – approximately 10% less per year than Soliris.