Ratify a new treatment for rare blood diseases in adults

Approved by the US Food and Drug Administration "FDA" Today, the injection is calling"Ultomiris" It contains the active ingredient (Ravulizumab) For the treatment of adult patients with hypoglycemic nocturnal hemoglobin (PNH), A rare and potentially fatal blood disease.

Richard Bazdor, director of the Center of Excellence in Oncology of the Food and Drug Administration and director of the Bureau of Hematology, said: "The approval of today for the new injection"Ultomiris" This will change the way patients with rare blood diseases are treated. "

He continued: "Prior to this approval, was the only approved treatment for PNH Treatment is required every two weeks, which can put a burden on patients and their families in terms of cost, but new injectionsUltomiris Contains a new formula and patients need treatment only once every 8 weeks, without prejudice to their effectiveness. "

The nocturnal hemoglobin is called hypoproteinemia (PNH) A rare acquired disease leading to rupture or destruction of red blood cells (hemolysis), patients are absent from PNH A particular protein usually prevents red blood cells from destroying the patient's immune system.

The common adverse drug reactions reported by patients in the clinical trials were headache and upper respiratory tract infections. Health care providers are advised to exercise caution when giving Ultomiris For patients with another infection.