Have you ever heard of a biosimilar? A biosimilar is essentially a biologic that is highly similar in structure and function to a reference product. Although this might seem technical, it really means that biosimilars have, clinically speaking, no significant differences from the original biologic. The treatment is safe, effective, and basically the same – albeit for (usually) a lower cost. This can help get treatments into the hands of patients when they need it. In May 2024, the U.S. Food and Drug Administration (FDA) approved BKEMV (eculizumab-aeeb) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
BKEMV is a biosimilar to Soliris (eculizumab), a therapy used to treat PNH and anti-AQP4 antibody positive neuromyelitis optica spectrum disorder (NMOSD). The injectable complement inhibitor is designed specifically to address hemolysis (red blood cell destruction) in people with PNH and complement-mediated thrombotic microangiopathy in aHUS.
How does the treatment work? BKEMV, a monoclonal antibody, inhibits part of the complement system from activating. The complement system plays a role in your body’s immune response – and, in these two rare diseases, stops the body from breaking down and destroying red blood cells.
While BKEMV is generally considered safe and well-tolerated, it can cause side effects such as diarrhea, low red blood cell count, nausea and vomiting, urinary tract infections, fever, cough, swelling of the lower extremities, and abdominal pain. BKEMV, similarly to SOLIRIS, may also increase the risk of meningococcal infections. At this point in time, BKEMV will only be available through the BKEMV Risk Evaluation and Mitigation Strategy program.
What is Paroxysmal Nocturnal Hemoglobinuria (PNH)?
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disorder. Normally, your stem cells develop into white blood cells, red blood cells, and platelets. But in PNH, PIGA mutations cause PNH cells to rapidly multiply while other healthy cells die. This causes your body to produce dysfunctional and defective red blood cells that are especially susceptible to hemolysis (red blood cell destruction). The immune system begins to attack those red blood cells, leading to numerous symptoms. PNH results from aplastic anemia treatment in up to 30% of cases. Usually, PNH is fatal within 10~ years of diagnosis. Identifying therapeutic interventions could save lives. If you have PNH, you might show signs of dark or bloody urine, a high heart rate, fatigue, chest and abdominal pain, sexual dysfunction in males, difficulty breathing, and headache.
What is Atypical Hemolytic Uremic Syndrome (aHUS)?
Atypical hemolytic uremic syndrome is a rare kidney disease characterized by hemolytic anemia (low red blood cell count caused by red blood cell destruction), thrombocytopenia (low platelet count), and uremia (the kidney’s inability to filter and excrete wastes through urine). People are genetically predisposed to aHUS; the condition is then triggered by an environmental element such as the flu or pregnancy. As pregnancy is a trigger, women are believed to be more affected by aHUS than men. People with aHUS may show signs like lethargy and fatigue, irritability, hematuria (blood in the urine), high blood pressure, proteinuria (protein in the urine), headaches, double vision, and seizures. Right now, there is no cure for aHUS. But treatments do exist, ranging from blood and platelet transfusions to blood pressure drugs and plasma therapy.
