Introduction: Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) is a rare hematological disorder characterized by episodic hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. , with additional clinical manifestations including thrombosis thrombosis: (throm-BOE-suss) A blood clot (thrombus) that develops and attaches to a blood vessel. and bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… . The US FDA approved a complement factor D inhibitor, danicopan danicopan: VOYDEYA is a prescription medicine used along with ravulizumab or eculizumab to treat breakdown of red blood cells that takes place outside of blood vessels (extravascular hemolysis), in adults with paroxysmal nocturnal hemoglobinuria (PNH). What is VOYDEYA? VOYDEYA is a… (Voydeya™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on 29 March 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan.
Areas covered: We systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to 6 May 2024.
Expert opinion: Danicopan acts on the alternative pathway of the complement cascade, preferentially controlling C3 fragment-mediated extravascular hemolysis. Recommended dosage is 150 mg orally three times a day, which can be increased to 200 mg three times a day when necessary. Vaccination is required before administration to prevent infections by encapsulated bacteria. In a pivotal phase 3 trial ALPHA, danicopan significantly increased hemoglobin hemoglobin: A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body. levels compared to placebo placebo: A placebo is an inactive pill, liquid, or powder that has no treatment value. (p < 0.0001), 60% of patients experienced an increase in hemoglobin levels of at least 2 g/dL, compared to none in the placebo group (adjusted difference of 47%; p < 0.0001). Common adverse events during danicopan treatment include headache and upper respiratory tract infection.
Keywords: ACH-4471; Danicopan; Voydeya; extravascular hemolysis; paroxysmal nocturnal hemoglobinuria.